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It should not surprise us that even in parts of the genome where we dont obviously see a functional code (i.e., one thats been evolutionarily fixed as a result of some selective advantage), there is a type of code, but not like anything weve previously considered as such. And what if it were doing something in three dimensions as well as the two dimensions of the ATGC code? A paper just published in BioEssays explores this tantalizing possibility

Isnt it wonderful to have a really perplexing problem to gnaw on, one that generates almost endless potential explanations. How about what is all that non-coding DNA doing in genomes?that 98.5% of human genetic material that doesnt produce proteins. To be fair, the deciphering of non-coding DNA is making great strides via the identification of sequences that are transcribed into RNAs that modulate gene expression, may be passed on transgenerationally (epigenetics) or set the gene expression program of a stem cell or specific tissue cell. Massive amounts of repeat sequences (remnants of ancient retroviruses) have been found in many genomes, and again, these dont code for protein, but at least there are credible models for what theyre doing in evolutionary terms (ranging from genomic parasitism to symbiosis and even exploitation by the very host genome for producing the genetic diversity on which evolution works); incidentally, some non-coding DNA makes RNAs that silence these retroviral sequences, and retroviral ingression into genomes is believed to have been the selective pressure for the evolution of RNA interference (so-called RNAi); repetitive elements of various named types and tandem repeats abound; introns (many of which contain the aforementioned types of non-coding sequences) have transpired to be crucial in gene expression and regulation, most strikingly via alternative splicing of the coding segments that they separate.

Still, theres plenty of problem to gnaw on because although we are increasingly understanding the nature and origin of much of the non-coding genome and are making major inroads into its function (defined here as evolutionarily selected, advantageous effect on the host organism), were far from explaining it all, andmore to the pointwere looking at it with a very low-magnification lens, so to speak. One of the intriguing things about DNA sequences is that a single sequence can encode more than one piece of information depending on what is reading it and in which direction viral genomes are classic examples in which genes read in one direction to produce a given protein overlap with one or more genes read in the opposite direction (i.e., from the complementary strand of DNA) to produce different proteins. Its a bit like making simple messages with reverse-pair words (a so-called emordnilap). For example: REEDSTOPSFLOW, which, by an imaginary reading device, could be divided into REED STOPS FLOW. Read backwards, it would give WOLF SPOTS DEER.

Now, if it is of evolutionary advantage for two messages to be coded so economically as is the case in viral genomes, which tend to evolve towards minimum complexity in terms of information content, hence reducing necessary resources for reproductionthen the messages themselves evolve with a high degree of constraint. What does this mean? Well, we could word our original example message as RUSH-STEM IMPEDES CURRENT, which would embody the same essential information as REED STOPS FLOW. However, that message, if read in reverse (or even in the same sense, but in different chunks) does not encode anything additional that is particularly meaningful. Probably the only way of conveying both pieces of information in the original messages simultaneously is the very wording REEDSTOPSFLOW: thats a highly constrained system! Indeed, if we studied enough examples of reverse-pair phrases in English, we would see that they are, on the whole, made up of rather short words, and the sequences are missing certain units of language such as articles (the, a); if we looked more closely, we might even detect a greater representation than average of certain letters of the alphabet in such messages. We would see these as biases in word and letter usage that would, a priori, allow us to have a stab at identifying such dual-function pieces of information.

Now lets return to the letters, words, and information encoded in genomes. For two distinct pieces of information to be encoded in the same piece of genetic sequence we would, similarly, expect the constraints to be manifest in biases of word and letter usagethe analogies, respectively, for amino acid sequences constituting proteins, and their three-letter code. Hence a sequence of DNA can code for a protein and, in addition, for something else. This something else, according to Giorgio Bernardi, is information that directs the packaging of the enormous length of DNA in a cell into the relatively tiny nucleus. Primarily it is the code that guides the binding of the DNA-packaging proteins known as histones. Bernardi refers to this as the genomic codea structural code that defines the shape and compaction of DNA into the highly-condensed form known as chromatin.

But didnt we start with an explanation for non-coding DNA, not protein-coding sequences? Yes, and in the long stretches of non-coding DNA we see information in excess of mere repeats, tandem repeats and remnants of ancient retroviruses: there is a type of code at the level of preference for the GC pair of chemical DNA bases compared with AT. As Bernardi reviews, synthesizing his and others groundbreaking work, in the core sequences of the eukaryotic genome, the GC content in structural organizational units of the genome termed isochores increased during the evolutionary transition between so-called cold-blooded and warm-blooded organisms. And, fascinatingly, this sequence bias overlaps with sequences that are much more constrained in function: these are the very protein-coding sequences mentioned earlier, and theymore than the intervening non-coding sequencesare the clue to the genomic code.

Protein-coding sequences are also packed and condensed in the nucleus particularly when theyre not in use (i.e., being transcribed, and then translated into protein) but they also contain relatively constant information on precise amino acid identities, otherwise they would fail to encode proteins correctly: evolution would act on such mutations in a highly negative manner, making them extremely unlikely to persist and be visible to us. But the amino acid code in DNA has a little catch that evolved in the most simple of unicellular organisms (bacteria and archaea) billions of years ago: the code is partly redundant. For example, the amino acid Threonine can be coded in eukaryotic DNA in no fewer than four ways: ACT, ACC, ACA or ACG. The third letter is variable and hence available for the coding of extra information. This is exactly what happens to produce the genomic code, in this case creating a bias for the ACC and ACG forms in warm-blooded organisms. Hence, the high constraint on this additional codewhich is also seen in parts of the genome that are not under such constraint as protein-coding sequencesis imposed by the packaging of protein-coding sequences that embody two sets of information simultaneously. This is analogous to our example of the highly-constrained dual-information sequence REEDSTOPSFLOW.

Importantly, however, the constraint is not as strict as in our English language example because of the redundancy of the third position of the triplet code for amino acids: a better analogy would be SHE*ATE*STU* where the asterisk stands for a variable letter that doesnt make any difference to the machine that reads the three-letter component of the four-letter message. One could then imagine a second level of information formed by adding D at these asterisk points, to make SHEDATEDSTUD (SHE DATED STUD). Next imagine a second reading machine that looks for meaningful phrases of a sensitive nature containing a greater than average concentration of Ds. This reading machine carries a folding machine with it that places a kind of peg at each D, kinking the message by 120 degrees in a plane. a point where the message should be bent by 120 degrees in the same plane, we would end up with a more compact, triangular, version. In eukaryotic genomes, the GC sequence bias proposed to be responsible for structural condensation extends into non-coding sequences, some of which have identified activities, though less constrained in sequence than protein-coding DNA. There it directs their condensation via histone-containing nucleosomes to form chromatin.

Figure. Analogy between condensation of a word-based message and condensation of genomic DNA in the cell nucleus. Panel A: Information within information, a sequence of words with a variable fourth space which, when filled with particular letters, generates a further message. One message is read by a three-letter reading machine; the other by a reading machine that can interpret information extending to the 4thvariableposition of the sequence. The second reader recognizes sensitive information that should be concealed, and at the points where a D appears in the 4th position, it folds the string of words, hence compressing the sensitive part and taking it out of view. This is an analogy for the principle of genomic 3D compression via chromatin, as depicted in panel B: a fluorescence image (via Fluorescence In-Situ Hybridization FISH) of the cell nucleus. H2/H3 isochores, which increased in GC content during evolution from cold-blooded to warm-blooded vertebrates, are compressed into a chromatin core, leaving L1 isochores (with lower GC content) at the periphery in a less-condensed state. The genomic code embodied in the high-GC tracts of the genome is, according to Bernardi [1], read by the nucleosome-positioning machinery of the cell and interpreted as sequence to be highly compressed in euchromatin. Acknowledgements: Panel A: concept and figure production: Andrew Moore; Panel B: A FISH pattern of H2/H3 and L1 isochores from a lymphocyte induced by PHAcourtesy of S. Sacconeas reproduced in Ref. [1].]

These regions of DNA may then be regarded as structurally important elements in forming the correct shape and separation of condensed coding sequences in the genome, regardless of any other possible function that those non-coding sequences have: in essence, this would be an explanation for the persistence in genomes of sequences to which no function (in terms of evolutionarily-selected activity), can be ascribed (or, at least, no substantial function).

A final analogythis time much more closely relatedmight be the very amino acid sequences in large proteins, which do a variety of twists, turns, folds etc. We may marvel at such complicated structures and ask but do they need to be quite so complicated for their function? Well, maybe they do in order to condense and position parts of the protein in the exact orientation and place that generates the three-dimensional structure that has been successfully selected by evolution. But with a knowledge that the genomic code overlaps protein coding sequences, we might even start to become suspicious that there is another selective pressure at work as well

Andrew Moore, Ph.D.Editor-in-Chief, BioEssays

Reference:

1. G.Bernardi. 2019. The genomic code: a pervasive encoding/moulding ofchromatin structures and a solution of the non-coding DNA mystery. BioEssays41:12. 1900106

Here is the original post:
That Junk DNA Is Full of Information! - Advanced Science News

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces presentations at the upcoming 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, Florida. The two poster presentations will highlight clinical data from the Phase 1 study of RP-L102 utilizing Process B for the treatment of Fanconi Anemia (FA), as well as long-term follow-up data from the Phase 1/2 EUROFANCOLEN trial.

Details for Rockets poster presentations are as follows:Title: Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor CellsSession Title: Gene Therapy and Transfer: Poster IIPresenter: Sandeep Soni, M.D.Session Date: Sunday, December 8, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

Title: Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene TherapySession Title: Gene Therapy and Transfer: Poster IIIPresenter: Paula Ro, Ph.D.Session Date: Monday, December 9, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

The Sunday poster session will be followed by a breakout session to give investors and analysts the opportunity to ask questions and discuss the data. The breakout session, hosted by Rocket management, will be held on Sunday, December 8th at 8:30 p.m. EST, directly after Dr. Sonis presentation. At the event, Dr. Soni, Clinical Associate Professor of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine and principal investigator of the U.S. Phase 1 trial of RP-L102 and Paula Ro, Ph.D., Senior Scientist, Divisin de Terapias Innovadoras en el Sistema Hematopoytico, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundacin Jimnez Daz will be participating in a Q&A panel. For further information, please contact investors@rocketpharma.com.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natures gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

The rest is here:
Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 Process B for Fanconi Anemia at the 61st American Society of Hematology Annual...

-- On-Track to Initiate Potential Registration-Enabling Trials in MDS and DLBCL in 1Q 2020 ---- Entered into Collaboration with bluebird bio to Evaluate Antibody-Based Conditioning Regimen in Combination with LentiGlobin ---- Management to Host Conference Call at 8:00 a.m. ET Today --

MENLO PARK, Calif., Nov. 12, 2019 (GLOBE NEWSWIRE) -- Forty Seven, Inc. (Nasdaq:FTSV), a clinical-stage, immuno-oncology company focused on developing therapies to activate macrophages in the fight against cancer, today reported financial results for the third quarter ended September 30, 2019 and provided a business update.

In the third quarter, we continued to enroll patients in our Phase 1b clinical trial of magrolimab in myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), while preparing to initiate potential registration-enabling trials in MDS and diffuse large B cell lymphoma (DLBCL) in the first quarter of 2020, said Mark McCamish, M.D., Ph.D., President and Chief Executive Officer of Forty Seven. We have plans in place for both programs that we believe could enable us to pursue accelerated paths to approval and to address the unmet needs of substantial patient populations in need of safe, well-tolerated and effective new options.

Dr. McCamish continued, We also made important progress with our preclinical candidates, FSI-174 and FSI-189, and remain on track to advance both into clinical testing next year. This morning, we announced a new collaboration with bluebird bio to evaluate our antibody-based conditioning regimen, comprised of magrolimab and FSI-174, in combination with LentiGlobin. We believe this partnership will allow us to accelerate and expand our efforts to provide an alternative, antibody-only conditioning regimen that avoids chemotherapy/radiation exposure for patients undergoing hematopoietic stem cell (HSC) transplantation. We are excited to work with the bluebird team as we continue our efforts to fully exploit the CD47 pathway as a novel therapeutic target.

Third Quarter and Recent Business Highlights:

Magrolimab Clinical Programs:Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)

Non-Hodgkin Lymphoma (NHL)

Solid Tumors

FSI-174:

Key Upcoming Milestones:

Forty Seven will present expanded efficacy and durability data from the Phase 1b trial of magrolimab in combination with azacitidine in patients with MDS and AML in an oral presentation at the 61st American Society of Hematology (ASH) Annual Meeting, which will be held December 7-10, 2019 in Orlando, Florida. Also at ASH, Forty Seven will present a poster detailing preclinical data for FSI-174.

Additionally, the company expects to complete investigational new drug (IND)-enabling studies for both FSI-174 and FSI-189 before year-end.

Third Quarter 2019 Financial Results:

Conference Call Information:

Forty Sevenwill host a live conference call and webcast at8:00 a.m. ET today to discuss third quarter 2019 financial results and recent business activities. The conference call may be accessed by (866) 953-0780 (domestic) or (630) 652-5854 (international), and by referring to conference ID 7667736. A webcast of the conference call will be available in the Investors section of theForty Sevenwebsite at https://ir.fortyseveninc.com. The archived webcast will be available onForty Sevenswebsite approximately two hours after the conference call and will be available for 30 days following the call.

About Forty Seven, Inc.Forty Seven, Inc. is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways based on technology licensed from Stanford University. Forty Sevens lead program, magrolimab, is a monoclonal antibody against the CD47 receptor, a dont eat me signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in multiple clinical studies in patients with myelodysplastic syndrome, acute myeloid leukemia, non-Hodgkins lymphoma, ovarian cancer and colorectal carcinoma.

Forward-Looking Statements:

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as believe, continue, could, expect, "may," plan, potential, predict, "will," and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the timing of potential registration-enabling trials in MDS and DLBCL; the potential to pursue accelerated paths to approval of Forty Sevens clinical programs; the potential for Forty Sevens clinical programs to address unmet needs of patient populations; the timing of potential clinical trials in FSI-174 and FSI-189; the timing, acceleration and outcome of Forty Sevens collaboration with bluebird bio to provide an alternative, antibody-only condition regimen; the presentation of, timing of and outcome of results from thePhase 1b clinical trial evaluating magrolimab as a monotherapy and in combination with azacitidine for the treatment of MDS and AML; the timing of complete enrollment and acceleration in the Phase 1b clinical trial evaluating magrolimab as a monotherapy and in combination with azacitidine for the treatment of MDS and AML; the timing and outcome of a BLA filing; the Phase 1b/2 clinical trial of magrolimab in combination with rituximab for patients with relapsed/refractory NHL, including DLBCL;the timing of, enrollment in and outcome of the Phase 1b/2 clinical trial of magrolimab in combination with rituximab for patients with r/r NHL, DLBCL; the outcome of the evaluation of biomarkers for potential predictive value and the advancement into earlier lines of treatment; the timing of initial results from the Phase 1b trial of magrolimab in combination with avelumab in patients with ovarian cancer; the timing of initial results from the Phase 1b trial of magrolimab in combination with cetuximab in patients with colorectal cancer; the timing of a clinical trial to evaluate Forty Sevens antibody-based conditioning regimen, comprised of FSI-174 and magrolimab, with bluebirds LentiGlobin gene therapy platform for the treatment of beta thalassemia and sickle cell disease; the sufficiency of a single-arm trial evaluating efficacy and durability to support the registration of magrolimab in combination with azacitidine in patients with MDS and AML; the timing of and quality of results from investigational new drug-application enabling studies for FSI-174 and FSI-189 and their respective potential for approval by the FDA; the sufficiency of a single-arm pivotal study evaluating ORR and durability to support the registration of magrolimab in combination with rituximab in patients with r/r DLBCL; Forty Sevens ability to fund its clinical programs and the sufficiency of its cash and short-term investments; and Forty Sevens financial outlook.

Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward looking statements. The potential product candidates that Forty Seven develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all. In addition, clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release. Such product candidates may not be beneficial to patients or successfully commercialized. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Forty Seven's stock price. Additional information concerning these and other risk factors affecting Forty Seven's business can be found in Forty Seven's periodic filings with the Securities and Exchange Commission at http://www.sec.gov. These forward-looking statements are not guarantees of future performance and speak only as of the date hereof, and, except as required by law, Forty Seven disclaims any obligation to update these forward-looking statements to reflect future events or circumstances.

For more information please visit http://www.fortyseveninc.com or contactinfo@fortyseveninc.com.

For journalist enquiries please contact Sarah Plumridge atfortyseven@hdmz.comor phone (312) 506-5218.

For investor enquiries please contact Hannah Deresiewicz at Stern Investor Relations Inc. athannah.deresiewicz@sternir.comor phone (212) 362-1200.

Forty Seven Inc.Condensed Statements of Operations and Comprehensive Loss(Unaudited)(In thousands, except share and per share data)

Forty Seven Inc.Condensed Balance Sheets(in thousands)

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Forty Seven, Inc. Reports Third Quarter 2019 Financial Results and Recent Business Highlights - GlobeNewswire

12 November 2019

Imagine a place where it's not one baby born every minute, it's 4 every second.

Parenting on Female First

You don't need to imagine too hard, that's the world we live in. The popular TV show has it firmly ingrained into the nation's conscious that there's only "one born every minute". But did you know it's more like 4 born every second around the globe, that's 265 every minute?

The global population currently stands at 7.7 billion, and were adding to it by around 265 babies per minute. At this rate, the population is expected to continue to rise and to hit 8 billion in 2023 and 10 billion by 2050. There are around 130 million babies born every year around the globe. A big number like that means very little to most people; when you watch the global birth rate counter designed by cord blood collection and storage company Smart Cells, the number is far easier to visualise.

On opening the page, a counter starts charting birth rates on each continent by the second in real time, highlighting how in some areas, such as Sub-Saharan Africa, there is one baby born more frequently than every second: 72 per minute. The continent with the lowest birth rate might surprise you: North Americas total population is a figure not to be laughed at - almost 362 million - yet its birth rate is surprisingly small: just 11.64 babies are born to every 1000 members of the population, compared to 22.22 in the Middle East and North Africa, whose population numbers 444 million, the next closest population size.

These figures might be of interest to many, but what is more likely to be a shock is the impact that numbers like these have on pregnant women and new mothers around the world. Access to prenatal care and skilled staff present at births is dwindling in South Asia and Sub-Saharan Africa, where just 58% of births in the latter are attended by skilled healthcare professionals and only 79% of women in the former receive prenatal care. This compares to 99% and 100% respectively in North America. This accounts to millions of pregnant women around the world who do not have access to suitable care for their needs, and 50% of women globally who do not receive the level of care that is recommended during pregnancy. It is suggested that, partly due to inadequate care during delivery, an estimated 303,000 mothers and 2.5 million newborns died in the first month of life in 2017.

Highlighting these figures is real time is important in helping people understand the effect that the disparity in healthcare has in less developed countries. One of the most shocking figures the counter presents is how closely the percentage of children with anaemia seems to relate to lack of access to prenatal healthcare. In areas like Sub-Saharan Africa where 60% of children under 5 suffer from anaemia, just 58% of these births were attended by skilled health staff; in South Asia, the number stands at 55% of children with anaemia and only 79% of pregnant women having access to prenatal care. Anaemia is the most common all blood conditions but Shamshad Ahmed, CEO at Smart Cells, explains, Stem cell therapy can help alleviate the symptoms of anaemia by boosting the production of healthy red blood cells.

He continues:

Advances in medicine, sanitation, and food production, has helped population numbers increase faster from the 1900s onwards. However, experts do believe population growth will peak in the next 100 years. Recently the negative effect that higher population has on our planet has been highlighted by campaigners, celebrities and the Royal Family. Experts believe limiting your family to only one or two children can help stabilise population growth to a level that wont put as much strain on our planets natural resources. It's hard to imagine the global population and births until you see it in numbers.

The rest is here:
Watch the numbers rise - four babies born every second! - FemaleFirst.co.uk

NEW YORK / ACCESSWIRE / November 11, 2019 / Kadmon Holdings, Inc. (KDMN) today announced positive topline results from the planned interim analysis of ROCKstar (KD025-213), the fully enrolled pivotal trial evaluating KD025 in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy. The trial met the primary endpoint of Overall Response Rate (ORR) at the interim analysis, which was conducted as scheduled two months after completion of enrollment.

KD025 showed statistically significant ORRs of 64% with KD025 200 mg once daily (QD) (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (BID) (95% CI: 54%, 78%; p<0.0001). KD025 has been well tolerated and adverse events have been consistent with those expected in the patient population.

"We are extremely pleased with the outcomes of the interim analysis, which showed that KD025 has already greatly exceeded the threshold for success in this pivotal trial," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to sharing these results with the FDA at a pre-NDA meeting, where we will also discuss the timing for a regulatory filing for KD025 in cGVHD, which we expect to occur in 2020, subject to FDA input."

"KD025 was shown to be a highly active and well-tolerated therapy across the spectrum of this complex, multi-organ disease," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and a KD025-213 study investigator and Steering Committee member. "The response rates observed are particularly impressive since this study is being conducted in a real-world population with severe disease, supporting the potential role of KD025 in cGVHD patients who are in need of effective and well-tolerated therapies."

"It is highly encouraging to see the positive results from the pivotal trial are in line with those observed in the earlier Phase 2 study of KD025 in this difficult-to-treat disease," said Madan Jagasia, MD, Vanderbilt University, an investigator of the KD025-208 and KD025-213 studies and the KD025-213 Steering Committee chair. "These latest KD025 data continue to underscore the value that KD025 may offer to cGVHD patients."

KD025-213 is an ongoing open-label trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg QD or KD025 200 mg BID, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%, which was achieved in both arms of the trial at the interim analysis.

While the ORR endpoint was met at the interim analysis, the primary analysis of the KD025-213 study will occur in the first quarter of 2020, six months after completion of enrollment. This analysis will include updated safety data and efficacy data, including ORRs and secondary endpoints, such as duration of response, changes in corticosteroid dose and changes in quality of life. Kadmon plans to submit results from the KD025-213 study for presentation at an upcoming scientific meeting.

Conference Call and Webcast

Kadmon will host a conference call and webcast on Monday, November 11, 2019, at 5:00 p.m., Eastern time, to discuss the topline results of the interim analysis of the KD025-213 study.

To participate in the conference call, please dial (866) 762-3021 (domestic) or (703) 925-2661 (international) and reference the conference ID: 6468498. The accompanying slides will be available for download on Kadmon's website beginning at 5:00 p.m. Eastern time.

To listen online via webcast, please visit: https://edge.media-server.com/mmc/p/9b9w8p38. The webcast will be archived and will be available at http://investors.kadmon.com/presentations-and-events.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). KD025 was granted Breakthrough Therapy Designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy.

Story continues

About cGVHD

cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.

About Kadmon

Kadmon is a biopharmaceutical company developing innovative products for significant unmet medical needs. Our product pipeline is focused on inflammatory and fibrotic diseases as well as immuno-oncology.

Forward Looking Statements

This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, including KD025-213, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates, including KD025; (iv) the timing or likelihood of regulatory filings and approvals, including in connection with KD025-213; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel; (xx) our ability to achieve cost savings and other benefits from our efforts to streamline our operations and to not harm our business with such efforts; (xxi) the use of proceeds from our recent public offerings; (xxii) the potential benefits of any of our product candidates being granted orphan drug designation; (xxiii) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; and/or (xxiv) other risks and uncertainties. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in Kadmon's filings with the U.S. Securities and Exchange Commission (the "SEC"), including Kadmon's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and subsequent Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's website at http://www.sec.gov. Kadmon assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

Contact Information

Ellen Cavaleri, Investor Relations646.490.2989ellen.cavaleri@kadmon.com

SOURCE: Kadmon Holdings, Inc.

View source version on accesswire.com: https://www.accesswire.com/566116/Kadmon-Announces-that-KD025-Met-Primary-Endpoint-at-Interim-Analysis-of-Pivotal-Trial-in-Chronic-Graft-Versus-Host-Disease

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Kadmon Announces that KD025 Met Primary Endpoint at Interim Analysis of Pivotal Trial in Chronic Graft-Versus-Host Disease - Yahoo Finance

JERUSALEM & PARSIPPANY, N.J. & INCHEON, South Korea--(BUSINESS WIRE)--Teva Pharmaceuticals USA, Inc., a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), Celltrion, Inc., (KRX KRX:068270) and Celltrion Healthcare, Co., Ltd. (KRX KOSDAQ:091990), today announced that TRUXIMA (rituximab-abbs) injection is the first biosimilar to the reference product Rituxan1 (rituximab) now available in the United States with a full oncology label. TRUXIMA is currently indicated for the treatment of adult patients with non-Hodgkins Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL):

We are excited about the first FDA-approved biosimilar to rituximab in the U.S., stated Brendan OGrady, Executive Vice President and Head of North America Commercial at Teva. Tevas commitment to biosimilars is focused on the potential to create lower healthcare costs and increased price competition. This focus is consistent with Tevas mission of making accessible medications to help improve the lives of patients.

TRUXIMA was approved by the U.S. Food and Drug Administration (FDA) as the first rituximab biosimilar. The approval was based on a review of a comprehensive data package inclusive of foundational and extensive analytical characterization, nonclinical data, clinical pharmacology, immunogenicity, clinical efficacy, and safety data. In May 2019, the FDA approved TRUXIMA to match all of the reference products oncology indications for NHL and CLL. In light of a patent settlement with Genentech, Celltrion and Teva have a pending FDA submission for rheumatoid arthritis (RA), granulomatosis with polyangiitis (GPA), and microscopic polyangiitis (MPA), and a license from Genentech to expand the TRUXIMA label to include these indications in Q2 2020.

We are pleased to announce the launch of the first rituximab biosimilar, TRUXIMA, with our marketing partner Teva in the U.S. said Mr. Hyoung-Ki Kim, Vice Chairman at Celltrion Healthcare. We believe that the introduction of TRUXIMA into the U.S. market will contribute to addressing unmet needs of U.S. patients as well.

The Wholesale Acquisition Cost (WAC or list price) for TRUXIMA will be 10 percent lower than the reference product. TRUXIMA is being made available through primary wholesalers at a WAC of $845.55 for 100mg vial and $4227.75 for 500mg vial. Actual costs to individual patients and providers for TRUXIMA are anticipated to be lower than WAC because WAC does not account for additional rebates and discounts that may apply. Savings on out-of-pocket costs may vary depending on the patients insurance payer and eligibility for participation in the assistance program.

Dedicated patient support services are also available from Teva through the Comprehensive Oncology Reimbursement Expertise (CORE) program. CORE is available to help eligible patients, caregivers and healthcare professionals navigate the reimbursement process. CORE offers a range of services, including benefits verification and coverage determination, support for precertification and prior authorization, assistance with coverage guidelines and claims investigation, and support through the claims and appeals process. A savings program is also available for eligible commercially insured patients. To learn more, please visit TevaCORE.com. For healthcare professionals seeking additional information, there is also a dedicated site at TRUXIMAhcp.com.

Celltrion and Teva Pharmaceutical Industries Ltd. entered into an exclusive partnership in October 2016 to commercialize TRUXIMA in the U.S. and Canada.

Please see the Important Safety Information below including the Boxed Warning regarding fatal infusion-related reactions, severe mucocutaneous reactions, hepatitis B virus reactivation and progressive multifocal leukoencephalopathy. For more information, please visit the full prescribing information.

Important Safety Information

WARNING: FATAL INFUSION-RELATED REACTIONS, SEVERE MUCOCUTANEOUS REACTIONS, HEPATITIS B VIRUS REACTIVATION and PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY

Infusion-Related Reactions - Administration of rituximab products, including TRUXIMA, can result in serious, including fatal, infusion-related reactions. Deaths within 24 hours of rituximab infusion have occurred. Approximately 80% of fatal infusion-related reactions occurred in association with the first infusion. Monitor patients closely. Discontinue TRUXIMA infusion for severe reactions and provide medical treatment for Grade 3 or 4 infusion-related reactions

Severe Mucocutaneous Reactions - Severe, including fatal, mucocutaneous reactions can occur in patients receiving rituximab products

Hepatitis B Virus (HBV) Reactivation - HBV reactivation can occur in patients treated with rituximab products, in some cases resulting in fulminant hepatitis, hepatic failure, and death. Screen all patients for HBV infection before treatment initiation, and monitor patients during and after treatment with TRUXIMA. Discontinue TRUXIMA and concomitant medications in the event of HBV reactivation

Progressive Multifocal Leukoencephalopathy (PML), including fatal PML, can occur in patients receiving rituximab products

Warnings and Precautions

Infusion-Related Reactions - Rituximab products can cause severe, including fatal, infusion-related reactions. Severe reactions typically occurred during the first infusion with time to onset of 30-120 minutes. Rituximab product-induced infusion-related reactions and sequelae include urticaria, hypotension, angioedema, hypoxia, bronchospasm, pulmonary infiltrates, acute respiratory distress syndrome, myocardial infarction, ventricular fibrillation, cardiogenic shock, anaphylactoid events, or death.

Premedicate patients with an antihistamine and acetaminophen prior to dosing. Institute medical management (e.g. glucocorticoids, epinephrine, bronchodilators, or oxygen) for infusion-related reactions as needed. Depending on the severity of the infusion-related reaction and the required interventions, temporarily or permanently discontinue TRUXIMA. Resume infusion at a minimum 50% reduction in rate after symptoms have resolved. Closely monitor the following patients: those with pre-existing cardiac or pulmonary conditions, those who experienced prior cardiopulmonary adverse reactions, and those with high numbers of circulating malignant cells (>25,000/mm3)

Severe Mucocutaneous Reactions - Mucocutaneous reactions, some with fatal outcome, can occur in patients treated with rituximab products. These reactions include paraneoplastic pemphigus, Stevens-Johnson syndrome, lichenoid dermatitis, vesiculobullous dermatitis, and toxic epidermal necrolysis. The onset of these reactions has been variable and includes reports with onset on the first day of rituximab exposure. Discontinue TRUXIMA in patients who experience a severe mucocutaneous reaction. The safety of re-administration of rituximab products to patients with severe mucocutaneous reactions has not been determined.

Hepatitis B Virus Reactivation - Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure and death, can occur in patients treated with drugs classified as CD20-directed cytolytic antibodies, including rituximab products. Cases have been reported in patients who are hepatitis B surface antigen (HBsAg) positive and also in patients who are HBsAg negative but are hepatitis B core antibody (anti-HBc) positive. Reactivation also has occurred in patients who appear to have resolved hepatitis B infection (i.e., HBsAg negative, anti-HBc positive and hepatitis B surface antibody [anti-HBs] positive).

HBV reactivation is defined as an abrupt increase in HBV replication manifesting as a rapid increase in serum HBV DNA levels or detection of HBsAg in a person who was previously HBsAg negative and anti-HBc positive. Reactivation of HBV replication is often followed by hepatitis, i.e., increase in transaminase levels. In severe cases increase in bilirubin levels, liver failure, and death can occur.

Screen all patients for HBV infection by measuring HBsAg and anti-HBc before initiating treatment with TRUXIMA. For patients who show evidence of prior hepatitis B infection (HBsAg positive [regardless of antibody status] or HBsAg negative but anti-HBc positive), consult with physicians with expertise in managing hepatitis B regarding monitoring and consideration for HBV antiviral therapy before and/or during TRUXIMA treatment.

Monitor patients with evidence of current or prior HBV infection for clinical and laboratory signs of hepatitis or HBV reactivation during and for several months following TRUXIMA therapy. HBV reactivation has been reported up to 24 months following completion of rituximab therapy.

In patients who develop reactivation of HBV while on TRUXIMA, immediately discontinue TRUXIMA and any concomitant chemotherapy, and institute appropriate treatment. Insufficient data exist regarding the safety of resuming TRUXIMA treatment in patients who develop HBV reactivation. Resumption of TRUXIMA treatment in patients whose HBV reactivation resolves should be discussed with physicians with expertise in managing HBV.

Progressive Multifocal Leukoencephalopathy (PML) - JC virus infection resulting in PML and death can occur in rituximab product-treated patients with hematologic malignancies. The majority of patients with hematologic malignancies diagnosed with PML received rituximab in combination with chemotherapy or as part of a hematopoietic stem cell transplant. Most cases of PML were diagnosed within 12 months of their last infusion of rituximab.

Consider the diagnosis of PML in any patient presenting with new-onset neurologic manifestations. Evaluation of PML includes, but is not limited to, consultation with a neurologist, brain MRI, and lumbar puncture.

Discontinue TRUXIMA and consider discontinuation or reduction of any concomitant chemotherapy or immunosuppressive therapy in patients who develop PML.

Tumor Lysis Syndrome (TLS) - Acute renal failure, hyperkalemia, hypocalcemia, hyperuricemia, or hyperphosphatemia from tumor lysis, sometimes fatal, can occur within 12-24 hours after the first infusion of rituximab products in patients with NHL. A high number of circulating malignant cells (>25,000/mm3) or high tumor burden, confers a greater risk of TLS.

Administer aggressive intravenous hydration and anti-hyperuricemic therapy in patients at high risk for TLS. Correct electrolyte abnormalities, monitor renal function and fluid balance, and administer supportive care, including dialysis as indicated.

Infections - Serious, including fatal, bacterial, fungal, and new or reactivated viral infections can occur during and following the completion of rituximab product-based therapy. Infections have been reported in some patients with prolonged hypogammaglobulinemia (defined as hypogammaglobulinemia >11 months after rituximab exposure). New or reactivated viral infections included cytomegalovirus, herpes simplex virus, parvovirus B19, varicella zoster virus, West Nile virus, and hepatitis B and C. Discontinue TRUXIMA for serious infections and institute appropriate anti-infective therapy. TRUXIMA is not recommended for use in patients with severe, active infections.

Cardiovascular Adverse Reactions - Cardiac adverse reactions, including ventricular fibrillation, myocardial infarction, and cardiogenic shock may occur in patients receiving rituximab products. Discontinue infusions for serious or life-threatening cardiac arrhythmias. Perform cardiac monitoring during and after all infusions of TRUXIMA for patients who develop clinically significant arrhythmias, or who have a history of arrhythmia or angina.

Renal Toxicity - Severe, including fatal, renal toxicity can occur after rituximab product administration in patients with NHL. Renal toxicity has occurred in patients who experience tumor lysis syndrome and in patients with NHL administered concomitant cisplatin therapy during clinical trials. The combination of cisplatin and TRUXIMA is not an approved treatment regimen. Monitor closely for signs of renal failure and discontinue TRUXIMA in patients with a rising serum creatinine or oliguria.

Bowel Obstruction and Perforation - Abdominal pain, bowel obstruction and perforation, in some cases leading to death, can occur in patients receiving rituximab in combination with chemotherapy. In postmarketing reports, the mean time to documented gastrointestinal perforation was 6 (range 1-77) days in patients with NHL. Evaluate if symptoms of obstruction such as abdominal pain or repeated vomiting occur.

Immunization - The safety of immunization with live viral vaccines following rituximab product therapy has not been studied and vaccination with live virus vaccines is not recommended before or during treatment.

Embryo-Fetal Toxicity - Based on human data, rituximab products can cause fetal harm due to B-cell lymphocytopenia in infants exposed to rituximab in-utero. Advise pregnant women of the risk to a fetus. Females of childbearing potential should use effective contraception while receiving TRUXIMA and for 12 months following the last dose of TRUXIMA.

Most common adverse reactions in clinical trials of NHL (>25%) were: infusion-related reactions, fever, lymphopenia, chills, infection, and asthenia

Most common adverse reactions in clinical trials of CLL (>25%) were: infusion-related reactions and neutropenia

Nursing Mothers - There are no data on the presence of rituximab in human milk, the effect on the breastfed child, or the effect on milk production. Since many drugs including antibodies are present in human milk, advise a lactating woman not to breastfeed during treatment and for at least 6 months after the last dose of TRUXIMA due to the potential for serious adverse reactions in breastfed infants.

About TRUXIMA

TRUXIMA (rituximab-abbs) is a U.S. Food and Drug Administration (FDA)-approved biosimilar to RITUXAN (rituximab) for the treatment of adult patients with CD20-positive, B-cell NHL to be used as a single agent or in combination with chemotherapy or CLL in combination with fludarabine and cyclophosphamide (FC).

TRUXIMA has the same mechanism of action as Rituxan and has demonstrated biosimilarity to Rituxan through a totality of evidence.

About Celltrion Healthcare, Co. Ltd.

Celltrion Healthcare conducts the worldwide marketing, sales and distribution of biological medicines developed by Celltrion, Inc. through an extensive global network that spans more than 120 different countries. Celltrion Healthcares products are manufactured at state-of-the-art mammalian cell culture facilities, designed and built to comply with the US Food and Drug Administration (FDA) cGMP guidelines and the EU GMP guidelines.

About Celltrion, Inc.

Headquartered in Incheon, Korea, Celltrion is a leading biopharmaceutical company, specializing in research, development and manufacturing of biosimilar and innovative drugs. Celltrion strives to provide more affordable biosimilar mAbs to patients who previously had limited access to advanced therapeutics. Celltrion received FDA approval for TRUXIMA (rituximab-abbs) and HERZUMA (trastuzumab-pkrb) in 2018.

About Teva

Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) has been developing and producing medicines to improve peoples lives for more than a century. We are a global leader in generic and specialty medicines with a portfolio consisting of over 3,500 products in nearly every therapeutic area. Around 200 million people around the world take a Teva medicine every day, and are served by one of the largest and most complex supply chains in the pharmaceutical industry. Along with our established presence in generics, we have significant innovative research and operations supporting our growing portfolio of specialty and biopharmaceutical products. Learn more at http://www.tevapharm.com.

Teva's Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 regarding TRUXIMA, which are based on managements current beliefs and expectations and are subject to substantial risks and uncertainties, both known and unknown, that could cause our future results, performance or achievements to differ significantly from that expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to:

and other factors discussed in our Quarterly Reports on Form 10-Q for the first and second quarter of 2019 and in our Annual Report on Form 10-K for the year ended December 31, 2018, including in the sections captioned "Risk Factors and Forward Looking Statements. Forward-looking statements speak only as of the date on which they are made, and we assume no obligation to update or revise any forward-looking statements or other information contained herein, whether as a result of new information, future events or otherwise. You are cautioned not to put undue reliance on these forward-looking statements.

1 RITUXAN is a registered trademark of Genentech and Biogen.

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Teva and Celltrion Announce the Availability of TRUXIMA (rituximab-abbs) Injection, the First Biosimilar to Rituxan (rituximab) in the United States -...

Wall Street brokerages expect Crispr Therapeutics AG (NASDAQ:CRSP) to post earnings of $0.04 per share for the current quarter, according to Zacks. Three analysts have issued estimates for Crispr Therapeutics earnings, with the lowest EPS estimate coming in at $0.01 and the highest estimate coming in at $0.07. Crispr Therapeutics reported earnings per share of ($0.92) in the same quarter last year, which suggests a positive year-over-year growth rate of 104.3%. The business is scheduled to report its next quarterly earnings results on Monday, February 24th.

On average, analysts expect that Crispr Therapeutics will report full year earnings of $0.65 per share for the current financial year, with EPS estimates ranging from $0.58 to $0.71. For the next fiscal year, analysts expect that the company will post earnings of ($4.72) per share, with EPS estimates ranging from ($5.76) to ($3.67). Zacks EPS averages are a mean average based on a survey of research firms that cover Crispr Therapeutics.

Crispr Therapeutics (NASDAQ:CRSP) last issued its earnings results on Monday, October 28th. The company reported $2.40 earnings per share (EPS) for the quarter, topping analysts consensus estimates of ($0.95) by $3.35. Crispr Therapeutics had a negative return on equity of 2.60% and a negative net margin of 5.30%. The company had revenue of $211.93 million for the quarter, compared to analysts expectations of $6.32 million.

CRSP has been the topic of a number of recent research reports. Piper Jaffray Companies reiterated an overweight rating on shares of Crispr Therapeutics in a report on Monday, October 21st. Needham & Company LLC reiterated a buy rating and issued a $62.00 target price on shares of Crispr Therapeutics in a report on Wednesday, July 31st. TheStreet upgraded Crispr Therapeutics from a d rating to a c rating in a report on Monday, October 28th. Canaccord Genuity initiated coverage on Crispr Therapeutics in a report on Friday, July 26th. They issued a buy rating and a $72.00 target price on the stock. Finally, Zacks Investment Research cut Crispr Therapeutics from a hold rating to a sell rating in a report on Monday, September 30th. Two equities research analysts have rated the stock with a sell rating, four have assigned a hold rating and eleven have assigned a buy rating to the companys stock. Crispr Therapeutics has a consensus rating of Buy and a consensus target price of $57.95.

In other Crispr Therapeutics news, Director Pablo J. Cagnoni sold 7,500 shares of the firms stock in a transaction on Wednesday, October 30th. The stock was sold at an average price of $52.00, for a total transaction of $390,000.00. Following the transaction, the director now directly owns 7,500 shares in the company, valued at approximately $390,000. The sale was disclosed in a document filed with the SEC, which is available through this hyperlink. 21.40% of the stock is currently owned by company insiders.

A number of large investors have recently modified their holdings of the business. Nikko Asset Management Americas Inc. lifted its stake in Crispr Therapeutics by 48.4% during the 3rd quarter. Nikko Asset Management Americas Inc. now owns 2,777,414 shares of the companys stock valued at $113,846,000 after acquiring an additional 906,006 shares in the last quarter. ARK Investment Management LLC increased its position in shares of Crispr Therapeutics by 34.7% during the 2nd quarter. ARK Investment Management LLC now owns 2,724,349 shares of the companys stock valued at $128,317,000 after purchasing an additional 701,332 shares during the last quarter. Price T Rowe Associates Inc. MD increased its position in shares of Crispr Therapeutics by 19.6% during the 2nd quarter. Price T Rowe Associates Inc. MD now owns 738,869 shares of the companys stock valued at $34,801,000 after purchasing an additional 121,176 shares during the last quarter. Wells Fargo & Company MN increased its position in shares of Crispr Therapeutics by 23.9% during the 2nd quarter. Wells Fargo & Company MN now owns 603,905 shares of the companys stock valued at $28,443,000 after purchasing an additional 116,540 shares during the last quarter. Finally, Morgan Stanley increased its position in shares of Crispr Therapeutics by 6.1% during the 2nd quarter. Morgan Stanley now owns 284,984 shares of the companys stock valued at $13,423,000 after purchasing an additional 16,361 shares during the last quarter. Institutional investors and hedge funds own 51.09% of the companys stock.

Shares of CRSP traded up $2.67 during trading hours on Friday, reaching $53.58. The stock had a trading volume of 1,632,533 shares, compared to its average volume of 619,587. Crispr Therapeutics has a 12 month low of $22.22 and a 12 month high of $56.16. The firm has a 50-day simple moving average of $42.62 and a 200 day simple moving average of $44.25. The company has a current ratio of 8.32, a quick ratio of 8.32 and a debt-to-equity ratio of 0.06. The company has a market capitalization of $2.79 billion, a price-to-earnings ratio of -15.58 and a beta of 3.15.

Crispr Therapeutics Company Profile

CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9) gene-editing platform in Switzerland. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells.

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$0.04 EPS Expected for Crispr Therapeutics AG (NASDAQ:CRSP) This Quarter - Casper Courier

The University of Vermont Health Network, along with partners Invitae and LunaPBC, launched a pilot project on November 1, to offer the Genomic DNA Test as part of its clinical care. The test will provide information for 147 genes that are indicators of increased risk for certain diseases including hereditary cancers, cardiovascular conditions, and other medically important disorders for which clinical treatment guidelines are established. The test also screens for carrier status for other diseases.

Our overall health and longevity are determined about 30% by genetics, said Debra Leonard, M.D, Ph.D., chair, Pathology and Laboratory Medicine at UVM Health in a press release. But until now, most of our clinical health care decisions have been made without understanding the differences in each individuals DNA that could help guide those decisions.

The Genomic DNA Test will be offered over the next year to 1,000 patients who are over 18 years old, are treated by a UVM Health Network Family Medicine provider, are not pregnant, or the partner of someone who is currently pregnant, and are part of the OneCare Vermont Accountable Care Organization (ACO). The testing will be conducted by Invitae on healthy individuals who opt in to the pilot and will be provided with information about their potential risk of developing diseases like cancer or heart disease based on their genetic make-up, with the potential to adjust their healthcare and lifestyle to help mitigate some of these risks.

Nearly one-in-six healthy individuals exhibit a genetic variant for which instituting or altering medical management is warranted, said Robert Nussbaum, M.D., Invitaes chief medical officer in a prepared statement. Genetic screening like the Genomic DNA Test in a population health setting can help identify these risk factors so clinicians can better align disease management and prevention strategies for each patient.

The test and any pre- and post-test genetic counseling services will be provided to pilot project participants at no charge and results will become a part of each patients medical record and available to the patient and all of his or her healthcare providers.

In addition to providing patient-specific information that can help determine health and wellness decisions, patient genomic data can also be used in for broader research applications that are helping to unravel the genetic basis for a number of diseases.

Patients who are interested in making their data available for research purposes can share their data through LunaDNA, the sharing platform of pilot project partner LunaPBC. Patients who choose to share their data with researchers will become shareholders in LunaPBC, a public benefit corporation owned by the individuals who provide their genomic data to the company. Data provided by LunaDNA to researchers I de-identified to protect the privacy of its member-owners. In the future the patients will also be able to shareand receive additional LunaPBC share forlifestyle, nutrition and environmental data.

Vermonters who choose to share their genomic data for research will play a leading role in the advancement of precision medicine, said Dawn Barry, LunaPBC president and co-founder. This effort puts patients first to create a virtuous cycle for research that doesnt sacrifice patients control or privacy.We are proud to bring our values as a public benefit corporation and community-owned platform to this partnership.

According to UVM Health, the pilot program, run through the ACO is a step toward moving to a value-based healthcare system.

Vermont and other states are moving away from fee-for-service health care and toward a system that emphasizes prevention, keeping people healthy, and treating illness at its earliest stages, Leonard said. Integrating genetic risks into clinical care will help patients and providers in their decision-making.

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UVM Health Taps LunaPBC, Invitae on Genomic Testing Pilot Project - Clinical OMICs News

By Sandee LaMotte, CNN

(CNN) If certain vegetables have always made you gag, you may be more than a picky eater. Instead, you might be what scientists call a super-taster: a person with a genetic predisposition to taste food differently.

Unfortunately, being a super-taster doesnt make everything taste better. In fact, it can do the opposite.

Super-tasters are extremely sensitive to bitterness, a common characteristic of many dark green, leafy veggies such as broccoli, cauliflower, cabbage and Brussels sprouts, to name a few.

The person who has that genetic propensity gets more of the sulfur flavor of, say, Brussels sprouts, especially if theyve been overcooked, said University of Connecticut professor Valerie Duffy, an expert in the study of food taste, preference and consumption.

So that [bitter] vegetable is disliked, and because people generalize, soon all vegetables are disliked, Duffy said. If you ask people, Do you like vegetables? They dont usually say, Oh yeah, I dont like this, but I like these others. People tend to either like vegetables or not.

In fact, people with the bitter gene are2.6 times more likely to eat fewer vegetables than people who donot have that gene, according to a new study presented Monday at the annual meeting of the American Heart Association.

We wanted to know if genetics affected the ability of people who need to eat heart-healthy foods from eating them, said study author Jennifer Smith, a registered nurse who is a postdoc in cardiovascular science at the University of Kentucky School of Medicine.

While we didnt see results in gene type for sodium, sugar or saturated fat, we did see a difference in vegetables, Smith said, adding that people with the gene tasted a ruin-your-day level of bitterness.

Our sense of taste relies on much more than a gene or two. Receptors on our taste buds are primed to respond to five basic flavors: salty, sweet, sour, bitter and umami, which is a savory flavor created by an amino acid called glutamate (think of mushrooms, soy sauce, broth and aged cheeses).

But its also smelling through the mouth and the touch, texture and temperature of the food, Duffy said. Its very difficult to separate out taste from the rest. So when any of us say the food tastes good, its a composite sensation that were reacting to.

Even our saliva can enter the mix, creating unique ways to experience food.

When we come to the table, we dont perceive the food flavor or the taste of food equally, Duffy said. Some people live in a pastel food world versus others who might live in a more vibrant, neon food world. It could explain some of the differences in our food preference.

While there are more than 25 different taste receptors in our mouth, one in particular has been highly researched: the TAS2R38, which has two variants called AVI and PAV.

About 50% of us inherent one of each, and while we can taste bitter and sweet, we are not especially sensitive to bitter foods.

Another 25% of us are called non-tasters because we received two copies of AVI. Non-tasters arent at all sensitive to bitterness; in fact food might actually be perceived as a bit sweeter.

The last 25% of us have two copies of PAV, which creates the extreme sensitivity to the bitterness some plants develop to keep animals from eating them.

When it comes to bitterness in the veggie family, the worst offenders tend to be cruciferous vegetables, such as broccoli, kale, bok choy, arugula, watercress, collards and cauliflower.

Thats too bad, because they are also full of fiber, low in calories and are nutrient powerhouses. Theyre packed with vitamins A and C and whats called phytonutrients, which are compounds that may help to lower inflammation.

Rejecting cruciferous or any type of vegetable is a problem for the growing waistline and health of America.

As we age as a population, vegetables are very important for helping us maintain our weight, providing all those wonderful nutrients to help us maintain our immune system and lower inflammation to prevent cancer, heart disease and more, Duffy said.

Food scientists are trying to develop ways to reduce the bitterness in veggies, in the hopes we can keep another generation of super-tasters from rejecting vegetables.

Theres been some success. In fact, the Brussels sprouts we eat today are much sweeter than those our parents or grandparents ate. Dutch growers in the 90s searched their seed archives for older, less bitter varieties, then cross-pollinated them with todays higher yielding varieties.

People who already reject vegetables might try to use various cooking methods that can mask the bitter taste.

Just because somebody carries the two copies of the bitter gene doesnt mean that they cant enjoy vegetables, Duffy said. Cooking techniques such as adding a little fat, a little bit of sweetness, strong flavors like garlic or roasting them in the oven, which brings out natural sweetness, can all enhance the overall flavor or taste of the vegetable and block the bitterness.

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Attention Vegetable Haters: It Could Be In Your Genes - CBS Boston

After a class-action lawsuit was filed in October against several hospitals, Illinois Department of Children and Family Services (DCFS), and several doctors, audio has surfaced of some of the defendants in the case plotting to collude with DCFS to take children away from parents extra-lawfully. Recordings of these doctors at a committee meeting appear to bolster the plaintiffs' claims that the hospitals and agencies named in the suit "used the power given to them as State officials and/or employees and through their authorities and investigative powers to cause the Plaintiffs to be threatened and coerced into accepting unwanted and unnecessary medical procedures," as alleged in the lawsuit.

In April of 2018, the Perinatal Advisory Committee (PAC) that operates under the Illinois Department of Health met to discuss giving injections of Vitamin K in violation of the written refusals of parents. Not all the people on the recording can be identified by voice. PJ Media reached out to the members of the PAC but none would respond to identify who is speaking. But it is certain that all persons speaking are on the committee and a list of who was there can be seen at the end of this article. The following is a transcript of the recording.

WOMAN #2: On the wording on this and I actually just texted our neonatologist cause he wanted to be here and couldnt. We are a little concerned that its saying: The hospitals will. But it says: DCFS may. Mandating hospitals to do things but giving DCFS options to do things. So, then that puts the hospital in a little bit of a problem because I dont think hospitals want to be taking over custody all the time and then DCFS may investigate for medical neglect.

WOMAN #1: Its the issue is whether you are mandated to give vitamin k by taking protective custody. This is what this does. It doesnt mean that DCFS has to say the parent cant be the parent. But, it gives you the chance to give the child vitamin K through DCFS.

UNDETERMINED MAN: Correct.

WOMAN #1: So, I think it is okay. You dont need every parent to be accused of medical neglect and investigated. You need the right to give the vitamin k which DCFS will provide the custody for with this consistent message.

MS. LIGHTNER: I think you want the wiggle room of the may on the DCFS side because what I have heard is: If they are automatically slapped with medical neglect theres all sorts of ramifications there. So, you want DCFS to have that because if its shall

MAN #3: So, please clarify if DCFS says No, we are not going to investigate but the hospital has taken

WOMAN #1: You can take Protective custody is just the right to do what you think is right for the baby. And, DCFS, if they say, yes, that we agree with you, cause this is our rule. You give the vitamin K and then do any of us really care what happens next?

WOMAN #3: No, but can they sue you then?

WOMAN #1: No, because you have their you took protective custody. Thats the part that we have to assure with DCFS. That when we do this

MS. LIGHTNER: Need DCFS to assure you.

WOMAN #1: Yes, thats what I mean, DCFS has to say, This is our protocol, no matter what else we do: You are protected.

MAN #3: At what point does protective custody stop?

MULTIPLE VOICES. Right after

UNDETERMINED WOMAN: Its two minutes or whatever it is.

UNDETERMINED WOMAN: How much beyond?

UNDETERMINED MAN: As soon as you give the injection.

UNDETERMINED WOMAN Continues: Is it two minutes? Is it ten minutes? Do we wait until DCFS says we are coming or we are not coming?

WOMAN #1: They dont have to come. I think protective custody is you just claim that you have done it.

You can listen below.

This audio is shocking proof that doctors hold immense power over individual rights. Liberty died around a board room table in Chicago that day. Innocent parents had their children removed from their custody on nothing more than some unelected busybody's opinion that their medical degree was more important than constitutional rights and the right to informed consent.

At least one member of the PAC made it clear that she didn't care what happened after she imposed her will on American citizens using the power of the State. Although she may not care what happens next, when a doctor declares a parent unfit to make medical decisions and involves child welfare, the consequences are nothing short of horrific.

Medical kidnappings can and do result in accusations of "medical child abuse" by child welfare agents, leading to lengthy court battles and even the termination of parental rights. The Drake Pardo case in Texas illustrates this growing threat to families. Drake was taken from his parents and put into foster care because his mother wanted a second opinion on his condition. Theirs isn't the only story of doctors-gone-wild with power and professional privilege. The case of Justine Pelletier resulted in national attention when Boston Children's Hospital held a child with a rare mitochondrial disease for 16 months against her will, without proper treatment, and away from her parents in a psych ward until a judge intervened and ordered her to be returned to her family.

The epidemic of doctors taking custody of children because they deem themselves smarter and more capable of making decisions than parents is getting worse across all fields of medicine where children are seen, especially in rare genetic disorder cases. Mitochondrial Disease News reported the scary reality.

Hollinger, who has been with MitoAction for eight years, was previously a nurse at New Yorks Albany Medical Center. She spoke about medical child abuse at an October 2017 rare disease summit in Washington, D.C., sponsored by theNational Organization for Rare Disorders, which counts MitoAction among its 260 patient advocacy groups.

We need medical professionals, but the way I see it, the families are experts on their child in a way the doctor isnt, Hollinger explained. We are not all the same, even if we have the same genetic mutations.

She added: Child protective agencies are out there, and they work quite closely with the doctors. But theyre overworked and they know nothing about rare diseases. So, if some doctor or school says I think theyre overdoing it, Child Protective Services will ask the name of this disease. Theyre already aligning themselves and not in your ballpark.

The fact that doctors in Chicago are trying to expand the definition of medical neglect to include refusal of procedures that are not mandated by law, and DCFS appears to be eager to do their bidding, is unconscionable and if unchecked it will lead to more innocent families torn apart unnecessarily. The lawsuit is a good step forward to hold these people accountable, but there is no amount of money that can make right the damage that is done to a bonding newborn and mother when forcibly separated.

The recorded members of the PAC who were in attendance at the April 2018 meeting can be seen in the screenshot of the meeting minutes below.

Megan Fox is the author of Believe Evidence; The Death of Due Process from Salome to #MeToo. Follow on Twitter @MeganFoxWriter

See more here:
Doctors Caught on Tape Plotting to Take Custody of Newborns Whose Parents Refuse Vitamin K Shots | News and Politics - PJ Media

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that NHS Wales has accepted an offer for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.

This means that once the contract is finalized, patients with CF in Wales ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can access ORKAMBI (lumacaftor/ivacaftor) and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations can access SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor in the coming weeks.

The agreement also offers expanded access to KALYDECO (ivacaftor) to include those patients ages 12 months and older who have one of the nine licensed gating mutations.

Todays announcement is good news for the approximately 270 eligible cystic fibrosis patients in Wales who will soon have access to CFTR modulators to treat the underlying cause of their disease, said Ludovic Fenaux, Senior Vice President, Vertex International. We thank the authorities in Wales for their collaboration in accepting this offer under the same terms as were recently announced in England.

About CF in the UKOver 10,000 people in the UK have CF the second highest number in the world. Over 430 people in Wales have CF. CF is a debilitating, life-shortening inherited condition that causes progressive damage to organs across the body from birth. Currently, there is no cure for CF and half of people in the UK with CF die before they are 32. The daily impact of treatment is significant. It can take up to four or more hours, involving nebulizers, physiotherapy and up to 70 tablets a day. CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.

About ORKAMBI (lumacaftor/ivacaftor) and the F508del mutationIn people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftorSome mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

SYMKEVI is indicated for people with CF ages 12 and older who either have two copies of the F508del mutation or one copy of the F508del mutation and have one of the following 14 mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272-26AG, or 3849+10kbCT.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About KALYDECO (ivacaftor)KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

KALYDECO is indicated in people ages 12 months and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. KALYDECO is also indicated for the treatment of patients with CF ages 18 years and older who have an R117H mutation in the CFTR gene.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements by Mr. Fenaux in the fourth paragraph of this press release, statements regarding our expectations for the patient populations that will be able to access Vertexs medicines and the timing of such access, and statements about our expectations regarding a formal agreement in Northern Ireland. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Link:
Vertex Confirms Wales Offer Accepted for Access to All Licensed Cystic Fibrosis Medicines - Business Wire

LONDON--(BUSINESS WIRE)--

-Eligible patients in Northern Ireland will soon have access to ORKAMBI (lumacaftor/ivacaftor) and SYMKEVI (tezacaftor/ivacaftor), expanded access to KALYDECO (ivacaftor) under same terms as NHS England agreement-

Vertex Pharmaceuticals Incorporated (VRTX) today confirms that the Regional Pharmaceutical Procurement Service in Northern Ireland has accepted an offer for access to all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.

This means that once the contract is finalized, patients with CF in Northern Ireland ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene will have access to ORKAMBI (lumacaftor/ivacaftor) and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations will have access to SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor. We will support the arrangements being put in place to ensure clinicians will be able to prescribe to eligible patients within the next few weeks.

The agreement also offers expanded access to KALYDECO (ivacaftor) to include people ages 18 years and older who have the R117H mutation and those patients ages 12 months and older who have one of the nine licensed gating mutations.

We are pleased that the nearly 280 eligible cystic fibrosis patients in Northern Ireland will soon have access to CFTR modulators to treat the underlying cause of their disease and we thank the authorities in Northern Ireland for their collaboration and commitment in this agreement, said Ludovic Fenaux, Senior Vice President, Vertex International.

About CF in the UK Over 10,000 people in the UK have CF the second highest number in the world. Nearly 480 people in Northern Ireland have CF. CF is a debilitating, life-shortening inherited condition that causes progressive damage to organs across the body from birth. Currently, there is no cure for CF and half of people in the UK with CF die before they are 32. The daily impact of treatment is significant. It can take up to four or more hours, involving nebulizers, physiotherapy and up to 70 tablets a day. CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.

About ORKAMBI (lumacaftor/ivacaftor) and the F508del mutation In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

SYMKEVI is indicated for people with CF ages 12 and older who either have two copies of the F508del mutation or one copy of the F508del mutation and have one of the following 14 mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272-26AG, or 3849+10kbCT.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About KALYDECO (ivacaftor) KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

Story continues

KALYDECO is indicated in people ages 12 months and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. KALYDECO is also indicated for the treatment of patients with CF ages 18 years and older who have an R117H mutation in the CFTR gene.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements by Mr. Fenaux in the fourth paragraph of this press release and statements regarding our expectations for the patient populations that will be able to access Vertexs medicines and the timing of such access. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

View source version on businesswire.com: https://www.businesswire.com/news/home/20191112005777/en/

Go here to see the original:
Vertex Confirms Northern Ireland Offer Accepted for Cystic Fibrosis Medicines - Yahoo Finance

Rubin Jure

Director, Centro Privado de Neurologa y Neuropsicologa Infanto-Juvenil Wernicke

The ability to see plays a large role in the development of the brain so it makes sense that there would be a connection between vision and autism, which is essentially a condition of brain development.

When the eyes are open, vision is the dominant sense. Continuous input from the eyes links stimulation from other senses into a coherent whole.

A typical newborn already looks preferentially at faces, driven by an innate motivation to interact with others1. In this way, vision drives social-communication skills. Visual input shapes the brain during the first year of life. The visual system also processes aspects of nonverbal communication, such as shared visual attention, facial expressions, gestures and body postures.

Sight is also crucial for acquiring some basic concepts such as cause-and-effect relationships among actions, self as separate from others and object permanence.

So it stands to reason that without vision, brain development would go awry.

As a child neurologist, I treat children with various developmental disabilities, including autism. Every year, I see at least one blind child who shows the full clinical manifestations of autism. Because congenital blindness is rare, this was striking to me, particularly because the autism traits in these children are typically severe.

In 2012, I decided to investigate the relationship between autism and blindness in an unbiased population. Although the study was small, it indicated that autism is more than 30 times as common in blind people as in sighted people2. Other work from my team suggests this relationship is specific to vision: Hearing impairment is not strongly connected to autism3. The association is also independent of intellectual ability, showing that problems with cognition alone cannot explain the connection4.

The first report of autism in blind children appeared in more than 60 years ago5. In that study, the researcher identified autism in 5 of 60 infants who became blind because of retinopathy of prematurity, a condition in which the retina does not fully develop. (The rest of the children also had mild autism traits.)

Since then, scattered reports have drawn an association between congenital blindness and autism traits. But professionals typically did not recognize childrens social and other difficulties as autism often because they did not know much about autism. Instead they collectively called these issues blindism.

To a seasoned eye, the similarities between blindism and autism are striking. They include atypical communication, language and social skills, as well as stereotypies, resistance to change, severe anxiety and high pain tolerance. And like sighted autistic children, about one in four blind children experiences regression at 15 to 30 months of age.

To find out more about this connection, I visited a school for the blind and evaluated 38 of the schools 125 students for autism. I diagnosed autism in 18 of 25 students with congenital blindness but in only 1 of 13 with partial or acquired blindness. A statistical analysis showed that congenital blindness is the main factor responsible for the autism. No other variable, including etiology of blindness, the presence of intellectual disability, overt brain damage or socioeconomic status, accounted for its high prevalence2.

As part of this study, I evaluated 12 previous studies of blindness and autism. Each study focused on specific causes of blindness, under the assumption that the participants autism stemmed from the same cause say, congenital rubella or optic nerve atrophy. But I found that, taken together, the studies suggest that blindness itself (no matter what its cause) is connected to autism: About half of the collective 859 children who were blind from an early age also have autism. The rate of autism was even higher, ranging from 55 to 74 percent, in children with total congenital blindness.

Some researchers have proposed that the connection between autism and blindness is cognitive. Results published earlier this year suggest that idea is incorrect, however.

This population-based study looked at core autism traits in more than 3,000 adults with intellectual disability, 386 of whom are visually impaired. They found that the visually impaired adults with intellectual disability are more likely to have autism traits than the sighted adults, indicating that the effects of blindness extend beyond intellect. And once again, the prevalence of autism traits is highest among the adults with congenital blindness4.

My clinical experience jibes with the notion that intellectual disability is not what binds blindness and autism. There are highly intelligent people who are both blind and autistic. For example, I saw a congenitally blind teenage girl named Brisa who has social and communication difficulties as well as atypical prosody, the musical quality of speech. Brisa has excellent reasoning skills and excels academically.

Among the senses, vision also may have a special connection with autism. In 1991, we found that only 46 of 1,150 hearing-impaired children met the criteria for autism. The presence of autism is related more to medical conditions that affect the brain, such as congenital rubella or prematurity, than to the severity of the hearing impairment, suggesting that deafness itself does not contribute to autism3.

Researchers need to nail down the brain mechanisms that account for the autism-blindness overlap. One place to start looking is a brain region called the superior colliculus. This structure receives direct input from the retina. It is involved in not only the recognition of faces and biological movement but also the integration of sensory input with emotions, basic body functions and motor planning functions that are often altered in autism6.

Another outstanding question is: Why does a small proportion of congenitally blind children develop typically? Early in life, communication is mostly visual in nature, so how do some blind children acquire communication and social skills despite the lack of visual input? Understanding the factors that protect them could provide clues to autism therapies.

Rubin Jure is a child neurologist and director of the Centro Privado de Neurologa y Neuropsicologa Infanto-Juvenil Wernicke in Cordoba, Argentina.

See more here:
Seeing connections between autism and blindness - Spectrum

Just past the front desk of the Milwaukee Public Librarys Central Branch on West Wisconsin Avenue, theres a hallway you have to walk through to get to the stacks. Through the end of November, that hallway will be lined with a series of striking black and white photographic portraits.

The "My Heart Is Not Blind" exhibition is made up of photographs, a book, and audio recordings by Texas photographer Michael Nye. They tell the stories of people who have been affected by blindness or limited vision. Nye says he's been working on the project for seven years. The exhibition was brought to Milwaukee by Wisconsins Vision Forward, a nonprofit group that works with people who've experienced vision loss.

"This exhibit is about our shared humanity and our shared fragility," says Nye. "I think we all share both of those qualities in our lives. And its about adaptation and about perception, and its about understanding."

We met Nye at Milwaukee Public Library's Central Branch to see the exhibit and learn more about why he was compelled to tell these stories:

Photographer Michael Nye speaks with Bonnie North at the Milwaukee Public Library's Central Branch.

Read more here:
'My Heart Is Not Blind' Exhibition Explores Blindness And Perception - WUWM

In 2008, Aboriginal and Torres Strait Islander peoples had six times more blindness than other Australians. The leading cause of this blindness was unoperated cataract.

Compared with other Australians, Indigenous Australians were 12 times more likely to be left blind from cataract, had to wait more than 50 per cent longer for surgery and surgery was performed seven times less frequently.

These dire findings, along with some others, led to the development of the Roadmap to Close the Gap for Vision launched in 2012.

The Roadmap recognised that the pathway of care or the patients journey was complex like a leaky pipe with many leaks. But if only one or two of these leaks were fixed, the pipe would still leak.

As part of this work, the Roadmap identified 42 issues that needed addressing. It set out a long-term plan to provide well-coordinated care and support for Indigenous people requiring eye care.

Read more

It aimed for adequately resourced and supported eye services to meet the population-based needs of these communities.

The good news is that since 2012, some significant progress has been made with strong support from the Aboriginal Community Controlled Health sector, the eye care stakeholders and successive governments.

But more needs to be done.

The 2019 report on Indigenous Eye Health Measures by the Australian Institute of Health and Welfare (AIHW) shows that the number of outreach eye examinations received by Indigenous Australians has almost tripled in the last six years.

This is one of many changes that have occurred in the eye care system, including the organisation of regional networks to coordinate regional eye care, changes in Medicare item numbers, the enhancement of screening for diabetic retinopathy and the dramatic reductions in the rates of trachoma.

The 2019 Annual Update of the Roadmap shows that good progress is being made across the board. Twenty one of the 42 recommendations have now been fully implemented.

Progress is being made on every one of the intermediary steps and almost 80 per cent have been completed.

Read more

Progress has also been made in providing better access for cataract surgery. The number of cataract operations performed for Indigenous Australians has increased nearly two and a half times.

Its interesting that the AIHW data also shows that Indigenous people have cataract surgery at a much younger age than non-Indigenous people.

However, the 2015 National Eye Health Survey found only 59 per cent of Indigenous Australians who needed cataract surgery had actually had the surgery, whereas 88 per cent for non-Indigenous Australians had received surgery.

Overall, 73 per cent of all hospital admissions for Indigenous Australians are to public hospitals compared to 33 per cent for non-Indigenous Australians.

However, in Australia, some 70 per cent of cataract surgery is performed in private. Although the precise data is not available, it seems likely that most cataract surgery for Indigenous Australians occurs in public hospitals.

The average waiting time for Indigenous patients to have cataract surgery in a public hospital in 2016-17 was 58 per cent longer than that for non-Indigenous patients and they were also twice as likely to wait for more than one year.

Based on population size, we would expect about 7,581 cataract operations would be required in 2015-17 for Indigenous people, but AIHW reports only 5,131 operations were done that is 68 per cent of the estimated need.

Read more

This gap of 2,400 or so operations is very small when compared to the total of 296,570 cataract operations performed in 2017-18 in Australia.

Another problem that is so often overlooked is the waiting times for assessment of cataract in the eye clinic.

But, the data on outpatient waiting times is not easy to find and variably reported.

In Victorian hospitals, the median wait for an initial eye clinic appointment ranges from 62 days to 347 days 90 per cent of people would be seen between 159 and 828 days.

In South Australia, the median waiting time is between 12 and 18 months and the maximum waiting time is between 61 months to 134 months thats more than 11 years.

Clearly, the public hospital system is not working well in providing equitable and timely cataract surgery.

Given the propensity for Indigenous patients to seek public hospital care, this actually has a discriminatory effect, so its a key area that needs to be addressed if we really are going to close the gap for vision.

The Roadmap also included recommendations for regional planning and organisation, case management and support, resources to meet population-based needs, monitoring and evaluation and the need for oversight at multiple levels.

This list needs to be expanded to include some reform of the public hospital management of cataract surgery.

Read more

In March this year, the Council of Australian Governments Health Council set the elimination of avoidable blindness as a high priority issue for Aboriginal and Torres Strait Islander people and this also is one of the priorities in Australias Long Term National Health Plan.

However, to improve access to cataract surgery and close the gap for vision, a number of further changes are needed.

The regional stakeholder groups need their services fully funded to meet their population-based needs. People requiring assessment for cataract surgery should have access to public hospitals without long waits and, if that clinical assessment has to be done in private, it should be done without gap fees.

Then, once listed for cataract surgery, that surgery must be done in a timely manner.

Public hospitals should report on their waiting lists both for clinical assessment and for surgery. And a wait of less than 90 days should become the standard target for both the clinical assessment and the surgery waiting times.

Although a lot of progress has been made in improving eye care for Aboriginal and Torres Strait Islander people, there is still a gap in the eye care they receive and their eye health.

One of the critical areas remaining is the provision of prompt access to culturally safe and affordable cataract surgery.

It means much still needs to be done to rectify the often unacceptably long waiting times for both outpatient assessment and cataract surgery in Australias public hospitals.

Banner: Getty Images

See original here:
Can we close the gap on blindness by 2020? - Pursuit

We all are exposed daily to bisphenol-A (BPA) and other bisphenols estrogen-like substances added to food can liners, paper receipts and plastic containers.

That exposure, according to research that regulators are willfully ignoring, is increasingly linked to harmful health impacts ranging from birth defects to cancer.

A year-long investigation by Environmental Health News finds that the U.S. Food and Drug Administration has stacked the deck against such findings from independent scientists studying BPA as well as many compounds used in "BPA-free" products.

Hundreds of emails obtained via the Freedom of Information Act and dozens of interviews show that science is being perjured:

Significantly, the FDA's maneuvering to keep BPA unregulated extends a similar "get out of jail free" card to thousands of other suspected hormone-altering compounds.

"Their failure to use modern science in examining the risk of BPA and other bisphenols leaves the health of the American public at significant risk," said Pete Myers, founder and chief scientist at Environmental Health Sciences, which publishes Environmental Health News.

Environmental Health News is an award-winning nonpartisan organization dedicated to driving science into public discussion and policy. Read the four-part series below, as well as a comic strip interpretation of the investigation.

And follow the fallout from this investigation on Twitter at the hashtag: #ExposedBPA

Read more from the original source:
Exposed: How willful blindness keeps BPA on shelves and contaminating our bodies - Environmental Health News

Congenital vision loss may protect against psychosis because of the high stability of an internal world characterized by other sensory modalities, according to an article published in Schizophrenia Bulletin.

Dr Thomas A Pollack and Dr Philip R Corlett, researchers at Kings College London in the United Kingdom and Yale University in New Haven, Connecticut, respectively, used a Bayesian prediction error minimization model to illuminate the role of vision loss in psychosis, particularly regarding positive symptoms.

The Bayesian approach to psychosis in schizophrenia conceptualizes the brain as a hierarchicalinference machine: the brain interprets the probability of present events using information gleaned from past stimuli, or priors. The more precise a prior, the more strongly it influences decision making and beliefs at higher levels in the hierarchy. In schizophrenia, this hierarchy is disturbed, and irrelevant stimuli may become abnormally salient.[to] beliefs higher in the hierarchy.

Congenital blindness may protect against these computational deficits that underlie schizophrenia. Investigators hypothesize that blind individuals experience greater stability of high-level priors, possibly driven by increases in N-methyl-D-aspartate receptor (NMDAR)-mediated signaling. NMDAR-mediated signaling is thought to be top-down and modulatory in nature. Visual deprivation has been shown to cause higher-level, multisensory neurons to shift from sensory-driven responses to a more modulatory influence, a phenomenon that is likely NMDAR dependent.

According to the researchers, other published data suggest that visual deprivation causes increases in NMDAR-dependent cortical excitability. As such, increased top-down modulatory signaling associated with stable higher-level priors is more prevalent in the visually impaired brain. This computational rationale may explain the protective effect congenital blindness offers against psychosis. With more stable priors, blind individuals are less susceptible to the perception abnormalities that characterize schizophrenia.

Notably, later-life vision loss offers no such protective effect against psychosis. The top-down model cortical hierarchy asserts that the brain is equipped with hyperpriors, or internal expectations concerning perceived features of the world. In psychosis, a hyperprior may generate false attributions of the causes of sensory input. According to this same model, hyperpriors also predispose the developmentally typical brain to have visual hallucinations following visual deprivation because the brain expects a cause for sensory data. In the absence of visual information, the brain creates false external attributions, which manifest as hallucinations.

According to this model, investigators outlined several experimentally testable hypotheses, including the theory that congenitally blind individuals will show lessened psychosis-proneness compared with their sighted counterparts and lower psychotomimetic response to ketamine. The Bayesian error minimization model may be useful in further efforts to explore the nature of psychosis, vision loss, and visual hallucinations.

Reference

Pollack TA, Corlett PR. Blindness, psychosis, and the visual construction of the world [published online October 11, 2019]. Schizophr Bull. doi: 10.1093/schbul/sbz098

See more here:
How Does a Visual Construction of the World Influence Psychosis? - Psychiatry Advisor

Mona Minkarastood on a train platform in Johannesburg, South Africa, tapping at her phone in frustration. The GPS was malfunctioning and the devices automated voice kept repeating that there was no transit information available.

Minkara, a newly appointed assistant professor of bioengineering at Northeastern, has been blind since she was seven years old. She was in Johannesburg filming the first part of a documentary series demonstrating how she navigates public transportation around the world.

I always tell people I cant wait to get lost, Minkara says. Sometimes society tells you, Youre blind, so you cant do this. So my freedom matters so much to me.

In July, Minkara was awarded the Holman Prize by LightHouse for the Blind and Visually Impaired, which is given to individuals who are blind and want to push their limits with some sort of groundbreaking adventure. The award is named for James Holman, a blind, Victorian-era explorer who spent years traveling the world alone and successfully circumnavigated the globe.

As with Holman, Minkaras adventure is rooted in solo exploration. She started with a trip to Johannesburg in October. In December, she will fly to London, and explore Istanbul, Singapore, and Tokyo before returning home. She is traveling with a videographer, but the woman is not allowed to help her in any way other than by filming what happens.

The footage will be made into a five-episode documentary series called Planes, Trains, and Canes, which will be released on YouTube in 2020. Minkara intends the series to show how blind people deal with different public transportation systems, and that adventure is possible for anyone.

It gives me a sense of freedom, to be in a city that has good public transportation, Minkara says. It means I can do my own thing for myself. Thats huge.

At Northeastern, Minkara is using her background in computational chemistry to study molecules that reside on the inner surface of our lungs, called pulmonary surfactants. They reduce the surface tension of water, which allows our lungs to expand more easily, helping us breathe.

Minkara will be modeling this substance at the molecular level. Her work could help researchers understand how vaping affects our lung function, as well as lead to better treatments for diseases such as respiratory distress syndrome.

To do her research, Minkara works with access assistants who take notes, proof-read publications, and trace the shape of plots on the back of Minkaras hand, so she can understand what they look like. Their assistance is invaluable, Minkara says, but she hopes blind researchers will have more tools in the future, such as tactile plots or braille displays, that could provide tangible access to the different images they are working with.

Minkara, who grew up watching The Magic School Bus and reading stories of Sherlock Holmes, knew she wanted to be a scientist. Her blindness didnt change that goal.

I actually started out undergrad wanting to be a surgeon, she says with a laugh. I remember having a conversation with the pre-med advisor saying something like, Would you want a blind person cutting up your brain? And I thought, Hmm, maybe were not ready yet, as a society.

Instead, Minkara pursued computational chemistry. When she took a postdoctoral position at the University of Minnesota, her advisor, J. Ilja Siepmann, helped Minkara realize that her blindness was actually a strength in scientific research.

Siepmann pointed out that being blind had taught Minkara to think differently and solve problems in creative ways. He wanted her in his lab because those skills would help her approach research questions from different angles, and see things that a sighted person might miss.

It just floored me, Minkara says. It was the first time in my professional life in which somebody saw my blindness as an asset, when I had felt like I needed to keep on running to keep up with my peers.

And she envisions a future with a lot more blind researchers.

There are a lot of hurdles, but I personally feel like theyre worth overcoming, Minkara says. I want to be there for kids that are trying to be scientists and are blind. Or really, any kid that is trying to do something that society thinks they cant.

For media inquiries, please contact media@northeastern.edu.

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A bioengineering researcher who studies how vaping affects lung function sees a future with more blind scientists - News@Northeastern

Most blind people have some light perception but I'm one of the 2pc that have total darkness. I was born with retinoblastoma, an extremely rare cancer of the eye, and by four months old, both my eyes had been removed.

My parents had a fruit farm in Kildare and when I was born in the mid 1970s, there was no internet, no support groups.

I think they did not know what approach to take and so decided to treat me exactly the same as my younger twin sisters. They worked tremendously hard and everyone had to pitch in; blindness was no excuse. Their work ethic rubbed off on me, and I could not imagine a life without work.

The profit mentality

I left a secure and pensionable job with the National Council for the Blind (NCBI) to set up the Irish operations of Sight and Sound Technology, a 40-year-old company that provides hardware and software for the blind, visually impaired, and for people with learning and reading difficulties.

People told me I was insane and questioned would a British company remain in Ireland post-Brexit. But one year on, there are not enough hours in the day for me to keep up with the work.

I had worked in the technology area in the NCBI, so I knew there was a market here.

My main motivation is to provide a quality product that can help people, and I don't want to lose my values. It is hard to balance with the pressure that a profit has to be made for the shareholders.

Learning curve

The first two months were tricky. I had spent 17 years surrounded by colleagues and suddenly it was just me. I was walking 40 minutes to sit in an empty office and bang out emails to tell people who we were. I confided my difficulties to my boss and he told me I could arrange my working life any way I preferred.

So now I work from home, and come into the office when meeting people, and we have hired someone to help with admin. My mental health is better working from home. I go to a lot of events and travel quite a bit anyway, on average three times a month to the UK.

Usability is key

What we do is integrate specialist solutions into mainstream technology, to make it more accessible for the blind, visually impaired, and those with learning and reading difficulties.

I have a braille keyboard with the usual commands of a regular keyboard. I will either listen to my emails, or else use a refreshable braille display, that shows a series of pins raised and lowered. I can also plug in this braille display to my phone or my Kindle.

Zoom text magnification software and background readers are lifelines for those with impaired sight, and now applications read verbally from the screen in high-quality synthetic speech - which is crucial.

I have proved to be great quality control. Although I can read graphs in braille, I was finding that the most difficult part of my job was interpreting financial data with lots of figures and spreadsheets. Up until then, my colleagues did not have a blind person working as their equal, so it has been helpful in pinpointing where we need to improve usability. No matter how good technology is, it has to be easy to use, otherwise it's useless.

Advance schmoozing

I've learned that the trick with networking at conferences is to do most of my schmoozing beforehand. I will get a list of who is going and try to build a rapport by email before I go.

My phone is useful when looking for a person. I will ask at the desk to check who has registered and then can send a message to say I will be at this stand at 10.30am.

I'm really specific, but there is subtlety; I make it look seamless.

Otherwise, people say 'great, see you there', but I'm never going to spot someone across a room and I have to make it concrete.

Everyone has their own perceptions of their world as they know it. In an ideal world, all would be treated equally, and there is no doubt that society is getting better. The technology is improving, and getting cheaper, all the time and I'm hoping in time there will be more employment opportunities for the blind.

Compartmentalising

Myself and my fiance (Paralympian Nadine Lattimore) had our son Adam six months ago. This has thrown a grenade into my work-life balance; it is the biggest challenge I have faced and I have not yet got it right.

I do find it difficult to stop working and find myself sending emails with Adam on my lap. I just dislike putting off until tomorrow what I can do today.

It can be challenging for a blind person to find accessible fitness activities, but I've started walking more, with Adam strapped to my chest in the baby carrier.

Living your life

Nadine lost her sight 14 years ago and I do feel that growing up blind from birth, I have had an easier time of it.

My reality is normal for me. If I was offered sight back, I'm not sure what I would do. I think about this question a lot. It would be a huge disruption and throw my life right out of kilter.

People always ask how we manage with a newborn. But looking after a baby is such a tactile process; we have had no problems, it's been a joy. I know I have a higher risk of getting cancer again but I don't give it much thought; my life is to be lived now and it's a wonderful one.

Indo Business

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'Everyone had to pitch in on the farm, blindness was no excuse' - Independent.ie

The government won't say the words 'climate change' and 'fires' in the same sentence.

A great deal of NSW is currently on fire. It is Spring.

Usually that kind of environmental devastation begins, at worst, in Summer. So many blazes, particularly at this time of year, is worryingly out of the ordinary.

Of course, we all knew that something like this was coming. Weve been warned for decades that climate change was set to increase the number of freak weather events, and that the drought would continue to dry out our natural woodlands, making blazes inevitable.

But just try telling that to Australias politicians. After all, NSW Premier Gladys Berejiklian and Prime Minister Scott Morrison have spent every hour since the blazes began vehemently attempting to deny any link between the destruction and climate change.

According to them, its not right to politicise the blazes. Instead, we should simply mourn a problem while obstinately, repeatedly ignoring its causes.

Its enough to make you weep. Or, at least, thanks to those folks over at SBSsThe Feed, to weep while bitterly laughing.

Yep,The Feed have dusted off their best yellow wig, and filmed a sketch lampooning the governments almost pathological inability to say the words climate change and fire in the same sentence.

Beginning with an itemised list of the public figures that have gone on the record to draw the line between environmental collapse and our changing climate, the sketch descends into pure, hysterical surreality, as the governments obstinance slowly morphs into straight-up denial.

Watch it through your tears here:

Read the rest here:
NSW Fires: 'The Feed' Blasts Government's Blindness On Climate Change - Junkee