StemCell Therapy

PARIS--(BUSINESS WIRE)--Regulatory News:

Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announces that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LYS-GM101, the companys gene therapy candidate for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease. LYS-GM101 builds on Lysogenes extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.

The IND clearance follows the recent clinical trial authorization granted by the MHRA in the United Kingdom. Lysogene intends to initiate its global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The clinical trial will include a safety phase and a confirmatory efficacy phase. The company intends to dose a total of 16 patients, with dosage of the first patient expected in the first half of 2021.

We are very pleased to receive this IND clearance for LYS-GM101 which completes the MHRA approval received a few weeks ago. It represents a major milestone that marks our second CNS gene-therapy program to enter into a global clinical trial said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene. This IND clearance once again demonstrates our quality and timely execution, and our strong determination to bring new therapeutic solutions for diseases that currently have no treatment.

Christine Waggoner, President and Co-Founder of Cure GM1 Foundation added: Children with GM1 gangliosidosis represent a clear unmet medical need and we are thrilled to see a new therapeutic option entering the clinic, as it brings tremendous hope to families and the entire GM1 gangliosidosis community.

LYS-GM101 (adeno-associated viral vector serotype rh.10 expressing beta-galactosidase) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as Rare Pediatric Disease designation in the US in 2016.

Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors in infantile and juvenile GM1 gangliosidosis (NCT04310163).

About LysogeneLysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing. An adaptative clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

Forward Looking StatementThis press release may contain certain forward-looking statements, especially on the Companys progress of its clinical trials and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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Lysogene Receives FDA Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101 for the...

DUBLIN--(BUSINESS WIRE)--The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Market Insight, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the AAV Vector Based Gene Therapy, historical and forecasted epidemiology as well as the AAV Vector Based Gene Therapy market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The AAV Vector Based Gene Therapy market report provides emerging drugs, AAV Vector Based Gene Therapy market share of the individual diseases, current and forecasted AAV Vector Based Gene Therapy market size from 2017 to 2030 segmented by seven major markets. The Report also covers current AAV Vector Based Gene Therapy market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

The AAV Vector Based Gene Therapy epidemiology division provides the insights about historical and current AAV Vector Based Gene Therapy patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- AAV Vector Based Gene Therapy Epidemiology

Estimates show that the highest cases of AAV vector based gene therapy in the 7MM were in the United States, followed by Germany, Japan, France, the United kingdom, Italy, and Spain in 2017.

AAV vector based gene therapy Drug Chapters

Drug chapter segment of the AAV vector based gene therapy report encloses the detailed analysis of AAV vector based gene therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the AAV vector based gene therapy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Scope of the Report

AAV Vector Based Gene Therapy Report Insights

AAV Vector Based Gene Therapy Report Key Strengths

AAV Vector Based Gene Therapy Report Assessment

Reasons to Buy

Companies Mentioned

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/p3sdwo

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Business...

bluebird bios beta-thalassaemia gene therapy betibeglogene autotemcel (beti-cel) has not been recommended by the UKs National Institute of Health and Care Excellence (NICE) for use on the NHS.

Beti-cel, marketed as Zynteglo in Europe, is a gene therapy intended for the treatment of transfusion-dependent beta-thalassaemia (TDT) in people aged 12 years and older who do not have a beta0/beta0 genotype.

It is indicated for TDT patients when haematopoietic stem cell transplantation (HSCT) is appropriate but there is no suitable donor.

TDT is the most severe form of thalassaemia, a condition wherein an inherited faulty gene leads to the inability of the body to produce normally functioning haemoglobin.

People living with TDT require life-long blood transfusions every two to five weeks.

In its draft recommendations, NICE commented that the follow-up on people included in clinical trials of beti-cel was not very long, adding that the population included was small.

NICE also determined that there were uncertainties around the cost-effectiveness of beti-cel, with the estimate for the gene therapy considerably higher than what it usually deems an acceptable use of NHS resources.

We are extremely disappointed with NICES decision not to recommend betibeglogene autotemcel as a treatment option in the UK, said Romaine Maharaj, executive director, UK Thalassaemia Society.

We also feel disheartened that our patient experts were misquoted and used out of context and feel that NICE needs to rectify this. Having an option and the access to a potentially curable treatment is vital and should be offered to patients, she added.

Read more here:
bluebird bio's beta-thalassaemia gene therapy rejected by NICE - PharmaTimes

Having raised $150 million, Orchard hopes to expand its therapy pipeline into larger indications and says its HSC approach is not limited to the vector it uses.

The firm has said the funding will also aid the launch of gene therapy Libmeldy in Europe. Last year, the firm received approval for the one-time treatment which is used to treat children with metachromatic leukodystrophy (MLD).

Though often viewed as a single technology, the delivery mechanism, the vector, and the type of cell modified in gene therapy can conjure different approaches. Libmeldy is an ex vivo Hematopoietic Stem Cell (HSC) gene therapy. The company told us HSCs are central to Orchards other products in its pipeline.

Image/iStock: CIPhotos

It also asserted that despite the use of a lentiviral vector, HSC is the facilitating technology in its gene therapy as it is not limited to the vector used.

We insert a working copy of the gene into the genome of HSCs, and once engrafted, these genetically modified cells can lead to multiple corrected cell types in the blood stream including white blood cells, red blood cells, platelets, and tissue macrophages, SVP, Leslie Meltzer told BioProcess Insider. Importantly, the progeny of HSCs can migrate into multiple organ systems including the brain and GI tract.

The firm uses a lentiviral vector where genes can be inserted, deleted, or modified. Orchard told us lentiviral vectors are an appealing choice because of its ability to stably integrate the genome and be passed on to all the progeny.

HSCs are particularly appealing because of their intrinsic ability to self-renew which means that these cells serve as the repository of stem cells is expected for the lifetime of the individual, said Meltzer.

To expand into larger indications, Chemistry Manufacturing and Controls (CMC) will be used to improve efficiency and the firm tout manufacturing as a critical step to transforming the capabilities of HSC.

We are focused on improving the HSC gene therapy manufacturing process through important technology innovations, including a scalable stable vector producing cell line, transduction enhancing compounds and a fully closed, automated drug product process.

With growing clinical data available, the firm is confident HSC gene therapy has the potential to make a durable impact in devastating disorders of the central nervous system.

Continue reading here:
Orchard Therapeutics talks the benefits of HSC therapy - BioProcess Insider

Patients with Bacillus Calmette-Guerin (BCG)unresponsive, non-muscle invasive bladder cancer (NMIBC) carcinoma in-situ (CIS) who were treated with N-803 (also known as ALT-803) added to BCG Vaccine experienced promising response rates and a tolerable safety profile, according to data presented at the 2021 Genitourinary Cancers Symposium.1

The findings, taken from cohort A of the phase 2/3 QUILT-3.032 trial (NCT03022825), showed that at a median follow-up of 10.7 months, 71% of evaluable patients (n = 51/72) achieved a complete response (CR) at any time (95% CI, 59%-81%), meeting the primary end point of the study.

Moreover, investigators reported a 56% probability of maintaining a CR with this approach at 12 months (95% CI, 38.8%-70.3%). Among responders, the estimated median duration of CR was 19.2 months (95% CI, 7.3not reached [NR]). Notably, the majority of patients, or 87.5%, (n = 70/80) have not progressed to radical cystectomy.

N-803 is an IL-15 superagonist antibody cytokine fusion protein with significant binding capacity to the IL-2R beta and gamma and increased half-life of the Fc receptor, Karim Chamie, MD, MSHS, an associate professor in residence of urology and Society of Urologic Oncology Fellowship Director at the University of California, Los Angeles, explained during a presentation on the findings. This results in significant accrual of T cells and natural killer cells without upregulation of regulatory T cells.

Results from the phase 1 trial examining N-803 plus BCG indicated that all patients (n = 9) with high-risk NMIBC experienced a durable CR or no recurrence. All patients were disease free at 24 months. Based on these data, the FDA granted N-803 a fast track therapy designation in December 2019; this led to the launch of the phase 2/3 trial to further examine this approach.2

To be eligible for enrollment on the trial, patients had to have histologically-confirmed BCG-unresponsive CIS with or without Ta or T1. Investigators defined BCG-unresponsive disease as persistent or recurrent CIS within 12 months of being treated with BCG; at least 5 of 6 doses of initial induction course with at least 2 of 3 doses of maintenance treatment or at least 2 of 6 doses of a second induction course were required.

Eighty patients were enrolled to cohort A and they received 50 mg of BCG in combination with 400 g of N-803 intravesically weekly x6 induction or single re-induction x3 plus maintenance.

The primary end point of the study is CR at any time with a lower bound 95% confidence interval of 20% or greater. At least 24 of the 80 patients enrolled needed to have experienced a CR to meet this end point. Key secondary end points included duration of CR and CR rate at 6 and 12 months. Other end points included progression-free survival, time to cystectomy, cystectomy rate, overall survival, and CR rate at any time per Central Pathology Review.

The mean age of patients enrolled on the study was 72.5 years, with 84% of patients aged 65 years or older. The majority of patients were male (86%), and most patients were white (90%). Eighty-two percent of patients had an ECOG performance status of 0, while 18% had a status of 1.

The median time from last previous BCG dose to study entry was 6.2 months and the mean time was 8.0 months. Additionally, the mean time from last prior BCG dose to first detected recurrence was 3.6 months with a median time of 2.7 months. Moreover, the median time from first detected recurrence to study entry was 2.3 months with a mean time of 4.6 months. The majority of patients had CIS at first recurrence (69%), with 21% had CIS/Ta and 9% had CIS/T1.

This is a heavily pretreated cohort with the median number of transurethral resection of the bladder tumor being 5.0 [and] a median number of prior BCG instillations being 16.2, said Chamie. One hundred percent of patients received prior BCG. In addition to that, 78% of patients received additional therapy with BCG, including checkpoint inhibitors [3%], chemotherapy [59%], interferon [13%], and vicinium [3%].

N-803 demonstrated promising results compared with other FDA-approved agents or investigational therapeutics in the space, such as pembrolizumab (Keytruda), which yielded a 41% CR rate at any time and a median duration of response of 16.2 months in responders.3 Similarly, nadofaragene firadenovec (rAd-IFNa/Syn3) elicited a 53% CR rate at any time with a median duration of response of 9.7 months.4

N-803 had a smaller cohort, however, the CR rate at any time was much higher, at 71% compared with 41% and 53% for pembrolizumab and nadofaragene, [respectively], Chamie reported. Our median duration was also favorable, although this must be taken within the context of a shorter median follow-up of 10.7 months. The cystectomy-free rate of 88% also compares favorably, with only 1 of 10 patients demonstrating extravesical disease.

The most common any-grade treatment-related adverse effects (TRAEs) included dysuria (18%), hematuria (15%), and pollakiuria (14%). Although grade 3 or higher TRAEs were observed during the study, with grade 3 arthralgia, myalgia, and extremity pain each reported in 1% of patients. Serious AEs were also rare but included cardiac disease (1%), hematuria (1%), and adenocarcinoma of the colon (1%).

Notably, no treatment-related serious AEs, no immune-related systemic AEs, and no grade 5 TRAEs were reported. Moreover, no TRAEs led to treatment discontinuation.

This is an administration profile that is quite familiar to urologists and favorable as an intravesical agent, concluded Chamie.

References:

1. Chamie K, Chang S, Gonzalgo M, et al. Phase II/III clinical results of IL-15RFb superagonist N-803 with BCG in BCG-unresponsive non-invasive bladder cancer (NMIBC) carcinoma in-situ (CIS) patients (cohort A). J Clin Oncol. 2021;39(suppl 6):510. doi:10.1200/JCO.2021.39.6_suppl.510

2. ImmunityBio granted FDA breakthrough therapy designation for N-803 IL-15 superagonist in non-muscle invasive bladder cancer. News release. BioSpace. December 4, 2019. Accessed February 13, 2021. http://bit.ly/2ZgGAvM

3. Balar AV, Kamat AM, Kilkarni GS, et al. Pembrolizumab (pembro) for the treatment of patients with Bacillus Calmette-Gurin (BCG) unresponsive, high-risk (HR) nonmuscle-invasive bladder cancer (NMIBC): over two years follow-up of KEYNOTE-057. J Clin Oncol. 2020;38(suppl 15):5041-5041. doi:10.1200/JCO.2020.38.15_suppl.5041

4. Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet. 2021;22(1):P107-117. doi:10.1016/S1470-2045(20)30540-4

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Addition of N-803 Results in Efficacy, Safety in NMIBC CIS Unresponsive to BCG - Cancer Network

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvards Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled Deep diversification of an AAV capsid protein by machine learning.

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature, said Eric Kelsic, Ph.D., Dynos CEO and co-founder. We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity, added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

About CapsidMap for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The companys proprietary CapsidMap platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

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Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of...

A gene therapy costing 16 crore is the only shot of life for nearly 200 children with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic disease, in Karnataka.

Last month, the therapy Zolgensma was offered free to a 14-month-old baby from Bhatkal (Uttara Kannada) who was the lucky winner of a lottery through a compassionate access programme by Novartis, the Swiss drugmaker. This lottery is held once in two weeks for SMA children across the world and doctors at Baptist Hospital, that has a dedicated Paediatric Neuromuscular Service, are hoping more children will benefit.

The therapy is a one-time infusion that takes about an hour, Ann Agnes Mathew, Consultant Paediatric Neurologist and Neuromascular Specialist, at Baptist Hospital told The Hindu. The therapy was approved by U.S. regulators in May 2019 and has since then turned into a miracle drug for this rare disorder that destroys a babys muscle control.

SMA is a disease caused by loss of nerve cells, which carry electrical signals from the brain to the muscles. The protein needed for this signalling is coded by a gene for which everyone has two copies - one from the mother and the other from the father. A child develops this disorder only if both the copies are faulty. Without treatment, this disease is ultimately fatal, said Dr. Mathew. The disease as it progresses, makes it extremely difficult for the babies to carry out basic activities like sitting up, lifting their head or swallowing milk.

Pointing out that the current treatment options range from medicines, which increase these proteins, to replacing the faulty gene, the doctor said, Zolgensma is a revolutionary treatment, which works by supplying a healthy copy of the faulty gene, which allows nerve cells to then start producing the needed protein. That halts deterioration of the nerve cells and allows the baby to develop more normally.

The drug has a 14-day shelf life and when it was sent from U.S. for the Bhatkal baby, it was stuck with customs for three days in mid-January making doctors jittery. Dr. Mathew said she had to personally meet the Customs officials to get it released. When we explained the situation to them, they immediately released it. Any further delay would have been risky. The parents have taken a house on rent and are staying near the hospital for follow up. The baby is doing fine now, she said.

Pointing out that 38 babies had succumbed to the rare disease in Karnataka in over one-and-a-half years, Dr. Mathew said most families have given up hope as they cannot afford the treatment.

The Paediatric Neuromuscular Service at Baptist Hospital is a pioneering centre in the country with a multidisciplinary team of a paediatric neurologist, paediatric neuromuscular specialist, paediatric geneticist, paediatric pulmonologist, paediatric intensivist, paediatric cardiologist and paediatric endocrinologist providing comprehensive care under one roof. This service is run in collaboration with Organisation for Rare Diseases India, a NGO.

A Bengaluru-based couple - Naveen Kumar and Jyothi - have taken to crowdfunding on ImpactGuru.com, a crowdfunding platform, to cover the cost of Zolgensma therapy for their 10-month-old baby Janish who was diagnosed with SMA.

Mr. Kumar, who works as an insurance surveyor and barely earns 30,000 a month, cannot afford the expensive treatment.

The couple were counting their babys milestones after his birth in February 2020. They caught his first smile and his first laugh but baby Janish never went past his first two milestones. The parents then rushed him to a pediatrician and from there the baby was referred to Baptist Hospital, said Dr. Ann Agnes Mathew, who has been treating the baby for the last five months.

Piyush Jain, co-founder and CEO, ImpactGuru.com, said over 22 lakh has been raised so far for baby Janish from over 1,500 donors.

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16 crore drug is the hope for SMA patients - The Hindu

Global Cancer Gene Therapy Market Report Provides Future Development Possibilities By Key Players, Key Drivers, Competitive Analysis, Scope, And Key Challenges Analysis. The Reports Conjointly Elaborate The Expansion Rate Of The Industry Supported The Highest CAGR And Global Analysis. This Report Providing An In Depth And Top To Bottom Analysis By Market Size, Growth Forecast By Applications, Sales, Size, Types And Competitors For The Creating Segment And The Developing Section Among The Global Cancer Gene Therapy Market . Market Expansion Worldwide With Top Players Future Business Scope and Investment Analysis Report

Global Cancer Gene Therapy Market Research Report Will Help To Take Informed Decisions, Understand Opportunities, Plan Effective Business Strategies, Plan New Projects, Analyze Drivers And Restraints And Give Vision On The Forecast. Report Is A Specialist And Broad Research Report On The Major Regional Market Conditions, Concentrating On The United States, China, Europe, Japan, Southeast Asia, And India Regions.

Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

Download Exclusive Sample Report (350 Pages PDF with All Related Graphs & Charts) @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-cancer-gene-therapy-market&pm

The major players covered in the cancer gene therapy market report are Adaptimmune, GlaxoSmithKline plc, bluebird bio, Inc, Merck & Co., Inc., CELGENE CORPORATION, Anchiano Therapeutics, Achieve Life Sciences, Inc among other domestic and global players.

Competitive Landscape and Cancer Gene Therapy Market Share Analysis

Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

This cancer gene therapy market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographical expansions, technological innovations in the market. To gain more info on Cancer gene therapy market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Cancer Gene Therapy Market Country Level Analysis

Cancer gene therapy market is analysed and market size insights and trends are provided by country, therapy and end user as referenced above.

The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

The country section of the cancer gene therapy market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Cancer gene therapy market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for cancer gene therapy market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the cancer gene therapy market. The data is available for historic period 2010 to 2018.

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Global Cancer Gene Therapy Market Insights, Size Estimation, Research Insights, COVID-19 Impact and Future Trends By 2028 KSU | The Sentinel...

Global chemotherapy induced peripheral neuropathy treatment marketis expected to gain market growth in the forecast period of 2021 to 2028. Data Bridge Market Research analyses the market is growing at a healthy CAGR in the above-mentioned research forecast period. Emerging markets and huge investments in research and development are the factors responsible for the growth of this market.

The analysis covered in the Chemotherapy Induced Peripheral Neuropathy Treatment marketing report gives an assessment of various segments that are relied upon to witness the quickest development amid the approximated forecast frame. The numerical and statistical data has been denoted in the graphical format for a clear understanding of facts and figures. This market report contains historic data, present market trends, environment, technological innovation, upcoming technologies and the technical progress in the related industry. It not only identifies the emerging trends along with major drivers, challenges and opportunities in the market but also analyses them efficiently.

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The major players covered in the chemotherapy induced peripheral neuropathy treatment market are Metys Pharmaceuticals, Nemus Bioscience, Sun Pharmaceutical Industries Pvt Ltd., PledPharma, Mylan N.V., GlaxoSmithKline, plc., Regenacy Pharmaceuticals, Pfizer, Inc., Asahi Kasei Pharma, Apexian Pharma, Solasia Pharma, Immune Pharmaceuticals, DermaXon, Krenitsky Pharmaceuticals among other domestic and global players.

Global Chemotherapy Induced Peripheral Neuropathy Treatment Market Scope and Market Size

The chemotherapy induced peripheral neuropathy treatment market is segmented on the basis of treatment, drug class, drug type, end-users and distribution channel. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

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Chemotherapy Induced Peripheral Neuropathy Treatment Market Country Level Analysis

The chemotherapy induced peripheral neuropathy treatment market is analysed and market size information is provided by country, treatment, drug class, drug type, end-users and distribution channel as referenced above.

The countries covered in the chemotherapy induced peripheral neuropathy treatment market report are U.S., Canada, Mexico in North America, Brazil, Argentina, Peru, Rest of South America, as part of South America, Germany, France, U.K.,Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Hungary, Lithuania, Austria, Ireland, Norway, Poland, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Vietnam, Rest of Asia-Pacific, Saudi Arabia, U.A.E, Egypt, Israel, Kuwait, South Africa, Rest of Middle East and Africa, as a part of Middle East and Africa.

On geographical estimation, North America accounts the largest market share due to the presence of high healthcare expenditure and favourable reimbursement policies for the treatment. Europe accounts the second largest market share due to increased genetic disorders and presence of refined medical facilities. Asia-Pacific is expected to account for the largest market share over coming years for the chemotherapy induced peripheral neuropathy treatment market due to constant rise in the incidence of cancer with increased demand for cost-efficient therapeutics.

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Global Chemotherapy Induced Peripheral Neuropathy Treatment Market Industry Perspective, Comprehensive Analysis, and Forecast 2028||Nemus Bioscience,...

Urine spot for albumin:creatinine ratio (UACR) values are higher in patients with uncontrolled type 2 diabetes (T2D) and in patients with a longer duration of disease, according to new research.

The findings bolster the case for annual UACR tests as a routine part of care, the authors concluded, since the tests can identify early signs of diabetic nephropathy.

Diabetic nephropathy is a complication of diabetes that is linked with chronic kidney disease leading to end-stage renal disease. Early detection of nephropathy can be a key factor in clinical outcomes. The presence of albumin in urine is associated with diabetic neuropathy. The American Diabetes Association recommends yearly screening for microalbuminuria. UACR is a more efficient method of testing for albumin, replacing the older standard of 24-hour urine collection.

Corresponding author Ayesha Malik, of Hameed Latif Hospital, in Pakistan, and colleagues noted that strict glycemic control can prevent microalbuminuria. The investigators sought to better understand how common microalbuminuria is among patients with T2D and the extent to which it correlates with diabetic control.

Malik and colleagues recruited 133 patients for their study, and recorded data such as body mass index, duration of diabetes, and hemoglobin A1C levels. The patients were given morning, mid-stream UACR tests, which were then evaluated for albumin.

The study population was mostly male (60.9%) and had an average age of 54.5 years. The mean duration of diabetes in the group was 6.29 years, and mean A1C was 8.99%.

Overall, 30.1% of patients had diabetic neuropathy, and 25.6% had microalbuminuria. In addition, 4.5% had macroalbuminuria.

The investigators then performed a Pearson product-moment correlation coefficient, which showed that high UACR correlated with a higher likelihood of uncontrolled diabetes and a longer duration of diabetes.

Some of the factors that influence kidney disease development include genetics, blood sugar control, and blood pressure, the authors said. The impact of strict diabetic control on prognosis is most pronounced in patients microalbuminuria. We found a significant correlation between higher HbA1c level and presence of microalbuminuria.

The investigators noted that T2D is growing in prevalence around the world, and they said local studies in the South Asian region have shown rates of microalbuminuria among patients with T2D that align with this new study.

However, due to poor healthcare infrastructure and lack of education, people are not routinely screened in developing or underdeveloped countries and end up presenting late in the disease course, they wrote.

Limitations of the study included the small sample size and the fact that the investigators did not take into account patients detailed drug histories or their histories of dietary protein intake prior to UACR. These factors could potentially alter UACR values.

Still, the investigators said their data show routine UACR testing can be an important component of kidney disease prevention.

Both early detection and progress of the disease can be determined by sensitive markers like the UACR, they concluded. In addition, our study shows that poorly controlled diabetes is associated with higher UACR. Involvement of the UACR in routine practice should be considered for all diabetic patients.

The study, "Prevalence of Microalbuminuria in Type 2 Diabetes Mellitus," was published online in Cureus.

Read more:
Microalbuminuria Is Higher in Patients With Uncontrolled Diabetes - MD Magazine

Fort Collins, Colorado: Chemotherapy Induced Peripheral Neuropathy Treatment Market Research is a precise analysis of the historical, current, and future phases of the market. The report was released specifically for business owners, executives, policymakers, and Chemotherapy Induced Peripheral Neuropathy Treatment stakeholders to determine likely market conditions. A few key aspects are highlighted in the Chemotherapy Induced Peripheral Neuropathy Treatment market report including raw materials, market strategies, production volumes, sales, and CAGR.

The report also sheds light on key market characteristics such as dynamics, viable market structure, global price volatility, supply and demand, market restraints, restraints, and growth drivers. The report also covers other important aspects like demand ratio, Chemotherapy Induced Peripheral Neuropathy Treatment price violations, strict rules, and unpredictable market fluctuations. It also highlights several growth drivers that may increase market returns in the near future.

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In-depth study of major segments of the Chemotherapy Induced Peripheral Neuropathy Treatment market:

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Chemotherapy Induced Peripheral Neuropathy Treatment Market also provides key insights into market segments that help players develop sound business strategies and deliver accurate products and services to their customers. He also helps market players and Chemotherapy Induced Peripheral Neuropathy Treatment business owners identify their potential buyers and grow their existing customer base so that they can better serve their customers. It also allows readers to plan specific directions and stay ahead of their competition.

Chemotherapy Induced Peripheral Neuropathy Treatment Market Segmentation, By Type

Chemotherapy Induced Peripheral Neuropathy Treatment Market Segmentation, By Application

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Leading Companies on the Chemotherapy Induced Peripheral Neuropathy Treatment market:

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Market Segments by Major Manufacturers:

Geographical scenario:

The geographical analysis of the Chemotherapy Induced Peripheral Neuropathy Treatment market has been done by examining different global regions such as North America, Latin America, Middle East, Asia-Pacific, and Africa on the basis of different parameters. The primary target for the Chemotherapy Induced Peripheral Neuropathy Treatment market is the Chemotherapy Induced Peripheral Neuropathy Treatment countries. The Chemotherapy Induced Peripheral Neuropathy Treatment market has broadly compiled through extensive research and analysis techniques such as qualitative and quantitative analysis. Furthermore, it offers a blend of SWOT and Porters five techniques to analyze the data of the global market. Moreover, this report offers a complete analysis of different business perspectives such as the ups and downs of the global market shares. To expand the market at the global level, it makes use of different techniques and sales methodologies for achieving the outcomes of the businesses.

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Following major key questions are addressed through this global research report:

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Chemotherapy Induced Peripheral Neuropathy Treatment Market Size 2021 | Covid-19 Impact Analysis | Business Outlook, Growth, Trends and Forecasts 2027...

Industry Growth Insights (IGI) recently published a report entitled, the Lebers Hereditary Optic Neuropathy Drug Market, describing the crucial aspects of the market by conducting an in-depth analysis of the current trend, emerging threats, and future market assessment. This report takes into account the adverse impact of the COVID-19 pandemic on the market for the period of 2020-2020 and provides a detailed information about how the market will perform during the forecast period, 2020-2027. Our research team presents the report in a simplistic manner supported by fact and actual figures that will assist clients to arrive an informed decision about their investment plans and business strategies.

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The report provides a holistic view of the market offering key insights of the market behavior over last four years and key assessment of the future market performance. It includes a systematic analysis of historical data for the period, 2015-2020 and draws upon assessment of the market performance for the forecast period, 2020-2027 by observing 2020 as the base year. With the reference to the available data, it provides vital insights on key factors such as drivers, restrains, trends, challenges, and opportunities of the Lebers Hereditary Optic Neuropathy Drug market growth.

This report provides a comprehensive outlook on the key segments and sub-segmentations that includes the complete information about the product types, applications, end-users and regions. It offers latest information regarding the growth rate, volume, and size of the market in respect to each segment and also explains the market performance of these segments in the respective five regions. Moreover, it discusses a wide range of the emerging market scope and potential drawbacks present in the segments.

Lebers Hereditary Optic Neuropathy Drug Market Report Includes:

The Global Lebers Hereditary Optic Neuropathy Drug Market Report is segmented into:

By Types:

ElamipretideGS-011IXC-201KH-176Others

By Applications:

HospitalClinicOthers

By Regions:

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The report covers the competitive landscape of various major global players, their current market positions, and key business strategies adopted to mark their major footprint in the market. This includes information about the product launch, expansion of the production facilities or plants, adoption of new technologies, latest merger & acquisition, partnership, and collaboration of the key players. It furthers provides concrete information about the existing market scope for the new entrants and the current competitive levels and scenario for the emerging players in the global market.

The Lebers Hereditary Optic Neuropathy Drug Market Report Covers the Following Companies:

Alkeus Pharmaceuticals, Inc.Biovista Inc.GenSight Biologics S.A.Ixchel Pharma, LLCKhondrion BVSpark Therapeutics, Inc.Stealth BioTherapeutics Inc.

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Regarding the methodology, the report is prepared by relying on primary and secondary sources including interviews of the company executives & representatives and accessing official documents, websites, and press release of the companies related to the Lebers Hereditary Optic Neuropathy Drug market. It also includes comments and suggestions from the experts in the market especially the representatives from government and public organizations as well as international NGOs. The report prepared by Industry Growth Insights (IGI) is known for its data accuracy and precise style, which relies on genuine information and reliable data source. Moreover, customized report can be available as per the clients wishes or specific needs. It takes into account of various research tools and methods including predictive analysis, Porters 5 force analysis, SWOT analysis, and real-time analytics.

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This report includes the estimation of market size in terms of value (USD) and volume (K MT), with applying top-down and bottom-up approaches to estimate and validate the overall scope of the market. The report is presented with a group of graphical representations, tables, and figures that shows a clear picture of the developments of the products and its market performance over the last few years. With this precise report, it can be easily understood the growth potential, revenue growth, product range, and pricing factors related to the Lebers Hereditary Optic Neuropathy Drug market. This report includes information on the latest government policies, norms, and regulations that can affect the dynamics of the market.

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Impact Of Covid-19 On Leber's Hereditary Optic Neuropathy Drug Market 2020: Remarking Enormous Growth With Recent Trends | Alkeus Pharmaceuticals,...

Cold weather can create health issues for many people, but some are more susceptible than others. In particular, cold can be especially difficult for cancer patients.

"Cancer, in general, puts a lot of stress on the body, and when we have stress on the body, we have trouble regulating other parts of our normal physiology," Dr. Nicholas Rohs, an oncologist at Mount Sinai in New York City, told AccuWeather.

Rohs said advising his patients about problems that can arise during cold weather is a regular part of his treatment routine.

It's not necessarily cancer itself that causes cold sensitivities -- it's therapies and side effects, Rohs said, that create problems, although cancer stresses out the entire system which puts a patient at a higher risk in general.

"People with really advanced cancers that are taking a really big toll on their body can have trouble regulating their own temperature," Rohs explained. "Inflammatory markers are flying around that can adjust how we are able to accommodate to our surroundings."

Chemotherapy and cold weather can be dangerous

Cold impacts can vary depending on the type of drug a patient is receiving. "Oxaliplatin is a very common chemotherapy medication we give," Rohs said. "That medication has a side effect called cold-induced neuropathy."

Patients being treated with Oxaliplatin who suffer cold-induced neuropathy can experience "numbness, tingling in their fingers, toes and even other parts of their bodies," Rohs said, adding that the condition "can get so bad they can be numb so they may not sense cold as well. But Oxaliplatin has this cold-induced neuropathy where when I give it to my patients they have hypersensitivity to cold to even a level where my patients say, 'I grabbed a sort of cold spoon and I couldn't hold it. I went to open my fridge and I couldn't.' And if it's really bad, people can be in an air-conditioned room and feel super uncomfortable because of it."

Story continues

Neuropathy is when the nerves malfunction, causing numbness or weakness. These patients experience cold temperatures, which can cause numbness, tingling, pain and muscle weakness. Neuropathies frequently start in the hands and feet but can affect other parts of the body as well. Rohs says this can cause them to be more prone to frostbite because they might not realize how cold their hands and fingers are getting in cold weather.

"If you have bad neuropathies -- and I actually have neuropathies not related to chemotherapy in my feet, I could go walk around in the snow for five or 10 minutes and not feel it the same way that a normal person would -- that can be really dangerous. If you can't sense the danger, you can't get away from it, so for people who have really significant neuropathies, they definitely need to be really thoughtful about wearing good cold-weather gear."

Rohs advises his patients to wear gloves and layers, so they can adjust to temperatures as they go in and out of buildings and different settings.

Julene Diedrich, a Marshfield Clinic nurse practitioner in Oncology tells Shine365, "We even tell these patients to avoid cold drinks or ice."

Cancer patients at higher risk of flu, cold, pneumonia and other viruses and infections

Chemotherapy can also compromise the immune system, making a patient extremely vulnerable to viruses or infections, which means common illnesses like the cold, flu or pneumonia can be life-threatening.

Rohs said someone who is not receiving chemotherapy might "easily fend off" a case of the common cold or flu, but "somebody who has a low blood count [a side effect of chemotherapy] might not be able to do the same. Patients receiving chemotherapy can also be susceptible to bacterial infection," he said. "They can get really robust pneumonia and things like that that they can't easily fight off."

One of the most common side effects of chemotherapy is that it attacks quickly dividing blood cells. Those cells include white and red blood cells. After a chemo treatment, patients often have lower white blood cell counts, particularly neutrophils, which are the first cells to arrive on the scene when someone experiences a bacterial infection. That's what lowers a patient's immunity. Having low red blood cell counts can make people feel colder and be more susceptible to cold.

"You don't have those red blood cells to circulate everything that you need and your body. If it's more anemic, your blood vessels in your peripheral part of your body -- the outsides of your body -- will clamp down more, so you can have more blood in your central areas because those are clearly more critical to us," Rohs said. "Our lungs, our heart -- all of these things are in the middle of our body."

So if the body is going into a state in which it's really cold, the circulatory system is going to shunt blood to the middle of our body, Rohs continued. "So when we're anemic, our body will try and keep most of the blood pool where it's most critical so you're likely to have cold fingers, toes, things like that."

Some patients can also be prone to cold-induced asthma, which occurs when lungs react to the temperature by narrowing, making it tougher to breathe.

"Patients who already have breathing issues, going out into the cold can be particularly irritating to their lungs and cause them to feel like their more short of breath and actually make them more short of breath because they can't breathe as well," Rohs said.

Cancer patients more prone to serious injury from an icy fall

In addition to treatment-induced issues, cancer patients often lose large amounts of weight, Rohs told AccuWeather. "Things like 20, 30 pounds for months so that's our biggest insulator," he said, adding that bodies that have lost a lot of weight due to chemotherapy can "have a lot of trouble regulating temperature."

Weight isn't just an insulator; it's padding that protects bones at a time when they are at their most fragile. Many cancer treatments cause bone loss also. Hormonal therapies often used to treat breast cancer can strip bone density, which is called osteoporosis. Osteopenia occurs when the body doesn't make new bone as quickly as it reabsorbs old bone. Both conditions put cancer patients at a high risk of becoming seriously injured when slipping and falling on ice. Rohs says a bone fracture can be devastating for a cancer patient whose body is already under tremendous stress.

"People have trouble adjusting to such an abrupt change in their physical health so they'll say, 'Well, I can still walk out to the street. Oh, it's a little icy, I do that all the time,' and they're more likely to fall and also when we talk about those neuropathies ...You might not be able to feel as steady on your feet, so you're more likely to slip and fall."

He added,"[If] we're having a snowstorm in New York I say. 'Hey -- stay home!' It's not the end of the world for many of my patients."

Bottom line, if you or someone you know is undergoing chemotherapy or other cancer treatments, pay particular attention to the weather forecast, so you can dress appropriately for the temperature.

"I tell my patients, you know, wear gloves, bring layers so that you can adjust to temperatures as you go in and out of buildings, or just different settings."

Keep checking back on AccuWeather.com and stay tuned to the AccuWeather Network on DirecTV, Frontier, Spectrum, Fubo, and Verizon Fios.

Read more from the original source:
What everyone should know about cancer and cold weather - Yahoo News

Journal Standard

Cold enough for you? This typical Midwest winter greeting pretty much sums up this weeks weather, but it should serve as a reminder, too it can be too cold.

As the temperature plunges and cold winds blow, so does the risk of frostbite. Skin exposed to extreme cold can develop frostbite in as little as five minutes, said FHN Podiatrist Roland Tolliver. Fingers in gloves and toes in boots can develop frostbite, too.

Though frostbite can happen to anyone, some conditions can make a person more susceptible: if you have diabetes, take blood thinners or smoke, your circulatory system may not work as efficiently or you may suffer from neuropathy, which means you have lost some feeling in your extremities. Poor circulation and neuropathy put you at higher risk for developing frostbite.

Skin that is cold and wet, like fingers in damp gloves and toes in wet socks, is even more susceptible to frostbite.

Danger Signs

Dr. Tolliver said one of the first signs of frostbite can be skin that is itchy, burnsor is numb. The skin also may turn white or greyish yellow, and as more damage occurs, feel waxy or hard. At its most severe, frostbite can cause blistering and even gangrene.

If you notice any of the signs of frostbite, go inside or get warmed up as quickly as possible.

Once you are in where its warm, remove any wet clothing and look closely at the affected area. You might see red skin and feel some pain or numbness. As you warm up, you may notice the area feels tingly that means the circulation is returning.

You can help speed the warming process by soaking the affected area in lukewarm not hot water. (The water temperature should be between 105-110 F.) Soak the area until the skin is back to its normal color and you dont feel any numbness.

A warm beverage like tea or cocoa can help you feel warmed up, too, but avoid any alcoholic beverages while your body is recovering from the cold. If you are feeling pain, an over-the-counter pain reliever may help.

If your pain increases or your skin doesnt return to its normal color, bundle up (in dry clothes leave the soggy gloves and boots behind) and head for the emergency department for medical attention.

Warm and dry

If youre planning to spend a lot of time outside in the cold, you probably know to wear long underwear and a turtleneck to help keep you warm.

Well-planned layers can help prevent frostbite, too: wear a thin pair of socks to pull moisture away from your feet underneath a thicker pair of socks for warmth. If you know youll be handling wet snow, wear waterproof gloves or layer pairs to keep your fingers as dry as possible.

Hand warmers and other gadgets to tuck in your pockets or shoes are easy to find this time of year, but people with poor circulation or who have lost some feeling in their extremities should be cautious using these items: if you cant feel your fingers or toes getting too cold, you likely wont be able to tell if they get too warm, either, and that can cause tissue damage, too.

The key to staying safe from frostbite, Tolliver said, is just to be aware of any signals your body may be sending. Take a warm-up break from shoveling or sledding when you need one with these temperatures, the snow isnt going anywhere!

Roland Tolliver, DPM, FACFAS, is a podiatrist at FHN. Hear more of his Dial-a-Doc interview on this topic at http://www.fhn.org/newsradio.asp.

Read more here:
FHN Health Column: Be careful in the cold protect your skin from frostbite - Freeport Journal-Standard

100 types of joint pain

Arthritis is an inflammation of the joints that can cause debilitating joint pain. There are more than 100 different kinds of arthritis and related conditions.

Arthritis affects more than 50 million adults and 300,000 children in America, according to the Arthritis Foundation. The causes and treatment options available vary from one type of arthritis to another.

To find the best treatment and management strategies, its important to determine the type of arthritis you have. Read on to learn about the types and what their differences are.

Osteoarthritis (OA), also called degenerative arthritis, is the most common type of arthritis. It affects about 27 million people in the United States, according to the Arthritis Foundation.

With OA, cartilage in your joints breaks down, eventually causing your bones to rub together and your joints to be inflamed with subsequent pain, bone injury, and even bone spur formation.

It may occur in just one or two joints, on one side of the body. Age, obesity, injuries, family history, and joint overuse can raise your risk of developing it. Common symptoms include:

To learn if you have OA, your doctor will take your medical history and conduct a physical exam. They may order X-rays and other imaging tests. They may also aspirate an affected joint, taking a sample of fluid from inside to check for infection.

Rheumatoid arthritis (RA) is a type of autoimmune disease in which your body attacks healthy joint tissue. The Arthritis Foundation estimates that about 1.5 million adults in the United States have RA. Nearly three times as many women have RA than men.

Common symptoms of RA include morning stiffness and joint pain, typically in the same joint on both sides of your body. Joint deformities can eventually develop.

Additional symptoms may also develop in other parts of your body including the heart, lungs, eyes, or skin. Sjgrens syndrome frequently occurs with RA. This condition causes severely dry eyes and mouth.

Other symptoms and complications include:

Your doctor cant use any single test to determine if you have RA. To develop a diagnosis, theyll likely take a medical history, conduct a physical exam, and order X-rays or other imaging tests.

Your doctor may also order a:

These tests can help your doctor learn if you have an autoimmune reaction and systemic inflammation.

Juvenile arthritis (JA) affects about 300,000 children in the United States have JA, according to the Arthritis Foundation.

JA is an umbrella term for several types of arthritis that affect children. The most common type is juvenile idiopathic arthritis (JIA), formerly known as juvenile rheumatoid arthritis. This is a group of autoimmune disorders that can impact childrens joints.

JIA starts to occur in children younger than 16 years old. It can cause:

Months of aching joints, swelling, stiffness, fatigue, and fevers may indicate juvenile idiopathic arthritis.

Other less common forms of JA include:

Ankylosing spondylitis (AS) and other types are autoimmune conditions that can attack the locations where tendons and ligaments attach to your bone. Symptoms include pain and stiffness, especially in your lower back.

Your spine will likely be affected the most, as AS is the most common of these conditions. It usually affects mainly the spine and pelvis but can affect other joints in the body.

Other spondyloarthropathies can attack peripheral joints, such as those in your hands and feet. In AS, bone fusion may occur, causing deformation of your spine and dysfunction of your shoulders and hips.

Ankylosing spondylitis is hereditary. Most people who develop AS have the HLA-B27 gene. Youre more likely to have this gene if you have AS and youre Caucasian. Its also more common in men than women.

Other spondyloarthritic diseases are also associated with the HLA-B27 gene, including:

Systemic lupus erythematosus (SLE) is another autoimmune disease that can affect your joints and many types of connective tissue in your body. It can also damage other organs, such as your:

SLE is more common among women, particularly those with African or Asian ancestry. Common symptoms include joint pain and swelling.

Other symptoms include:

You may experience more severe effects as the disease progresses. SLE affects people differently, but starting treatment to try to get it under control as soon as possible and working with your doctor can help you manage this condition.

Gout is a form of arthritis caused by the accumulation of urate crystals inside your joints. High levels of uric acid in your blood may put you at risk of gout.

An estimated 3.9 percent of American adults have gout thats 5.9 percent of American men and 2 percent of American women. Age, diet, alcohol use, and family history can affect your risk of developing gout.

Gout can be incredibly painful. A joint at the base of your big toe is most likely to be affected, although it can potentially affect other joints. You may experience redness, swelling, and intense pain in your:

An acute attack of gout can come on strong within a few hours during the course of a day, but the pain can linger for days to weeks. Gout can become more severe over time. Learn more about the symptoms of gout.

Infectious arthritis is an infection in one of your joints that causes pain or swelling. The infection can be caused by bacteria, viruses, parasites, or fungi. It can start in another part of your body and spread to your joints. This kind of arthritis is often accompanied by a fever and chills.

Reactive arthritis can occur when an infection in one part of your body triggers immune system dysfunction and inflammation in a joint elsewhere in your body. The infection often occurs in your gastrointestinal tract, bladder, or sexual organs.

To diagnose these conditions, your doctor can order tests on samples of your blood, urine, and fluid from inside an affected joint.

Up to 30 percent of those with psoriasis will also have psoriatic arthritis (PsA). Usually, youll experience psoriasis before PsA sets in.

The fingers are most commonly affected, but this painful condition affects other joints as well. Pink-colored fingers that appear sausagelike and pitting and degradation of the fingernails may also occur.

The disease may progress to involve your spine, causing damage similar to that of ankylosing spondylitis.

If you have psoriasis, theres a chance you could also develop PsA. If PsA symptoms start to set in, youll want to see your doctor to treat this as early as you can.

Many other forms of arthritis and other conditions can also cause joint pain. A few examples include:

If youre experiencing joint pain, stiffness, or other symptoms, talk to your doctor. They can help diagnose the cause of your symptoms and recommend a treatment plan. In the meantime, find relief from arthritis pain naturally.

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What Type of Arthritis Do You Have? Understanding Symptoms

Arthritis is a painful rheumatic condition that causes joint inflammation. There are many different types of arthritis that can affect the joints and other areas of the body and cause similar symptoms, such as pain, swelling, and stiffness.

There are more than 100 different types of arthritis. Some common types of arthritis include osteoarthritis (OA), rheumatoid arthritis (RA), and psoriatic arthritis (PsA).

Below, we explore these conditions, as well as other diseases that can present with arthritis. We also discuss potential treatment and when to seek medical guidance.

According to the Centers for Disease Control and Prevention (CDC), OA affects over 32.5 million adults in the United States.

Symptoms of OA include:

People often experience OA symptoms in the hips, hands, and knees.

The following increase the likelihood of developing OA:

Learn more about OA here.

RA is an autoimmune condition that causes painful swelling and inflammation in the joints. It typically affects the hands, wrists, and feet.

RA does not only affect joints. It can also cause problems in other organs of the body, including the heart, lungs, and eyes.

While some people experience a sustained progression of the disease, the intensity of symptoms usually comes and goes. Symptoms may include:

Another characteristic of RA is symmetrical involvement. This means pain and signs of inflammation occur on both sides of the body and in the same joints.

RA can cause lasting tissue damage, which can lead to:

Some people who have RA may also need assistance walking.

Some RA risk factors include:

Learn more about RA here.

PsA is an autoimmune condition.

Symptoms of PsA include:

PsA joint involvement is asymmetric, affecting different joints on either side of the body.

This type of arthritis can develop in people with a skin condition called psoriasis, which causes scaly, flushed, or silvery patches of skin. These patches can look different depending on a persons skin color.

Learn more about psoriasis on black skin here.

Researchers still do not fully understand what causes this form of arthritis. However, having a family history of PsA may increase a persons risk of developing this condition.

Learn more about PsA here.

Gout is a type of arthritis that causes painful swelling, often in a single joint at a time.

Symptoms of gout can flare up and go away quickly. They include:

Swelling is common in the big toe. Often, it also affects the knee or ankle joint.

A person may be at higher risk of developing gout if they:

Health conditions that may lead to gout include:

Gout may also occur due to metabolic syndrome, which is not a condition in itself. It refers to a number of characteristics, diseases, or habits that can make a person more likely to experience other health conditions, such as heart disease, stroke, or diabetes.

Learn more about gout here.

This chronic illness is an autoimmune condition that commonly affects females aged 1544 years.

Lupus is not a type of arthritis in itself. However, arthritis is one of the most common symptoms of this condition.

One symptom of lupus is the characteristic butterfly rash that can develop on the face. Other rashes can also develop on the arms, hands, and face. Rashes can worsen after sun exposure.

While symptoms may differ from person to person, they generally include:

Learn more about lupus here.

Juvenile arthritis, also known as childhood arthritis, affects children or even infants.

Symptoms of juvenile arthritis include:

There is no known cause of childhood arthritis. It appears to affect children regardless of race, age, or background.

Learn more about juvenile arthritis here.

Reaching a definitive diagnosis may take time, because many types of arthritis are similar or resemble other conditions.

Typically, a doctor will first check a persons medical and family history. They will also ask about symptoms and perform a physical exam. They may run tests such as:

Arthritis management depends on the type of arthritis. There are different forms of treatment available.

Medical treatment for arthritis may involve:

Surgery may not be necessary for everyone with arthritis. However, it can benefit certain complications of arthritis that result in malalignment of joints and functional limitations due to damaged joints. It can also help with intractable pain, which is when a person experiences pain that is difficult to treat or manage.

Common surgeries for arthritis include:

Physical therapy can be a great option for people with arthritis. It can help ease pain or increase activity.

Behavioral changes that can help with arthritis may include:

Some people find certain home remedies helpful in relieving pain and swelling from certain types of arthritis. These may include:

If a person has experienced joint symptoms that last more than 3 days, they should seek treatment from a healthcare professional.

Similarly, if a person has joint symptoms at different times within a month, they should also contact a doctor.

Medications and other treatments can help a person manage chronic symptoms, such as pain and swelling.

Pain will come and go with many types of arthritis. However, even if the pain resolves, a person should still seek treatment.

It is important to treat arthritis early. If left untreated, some types of arthritis may worsen over time and cause permanent disability.

Arthritis is a painful condition that causes joint inflammation.

Different types of arthritis can cause similar symptoms. It is vital to get the correct diagnosis, as it can help determine most effective treatment options.

The right treatment may also prevent future complications and help a person live a more active life.

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Common types of arthritis: Location, causes, treatment, and more - Medical News Today

February 9, 2021

After watching a close family member suffer with rheumatoid arthritis, Vidyanand Anaparti [PhD/15] was motivated to make a difference.

In 2015, Anaparti joined Max Rady College of Medicine professor Dr. Hani El-Gabalawys research program which focuses on discovering the origins of rheumatoid arthritis. Since then, Anapartis work has been recognized and was listed as one of the Arthritis Societys Top 10 Research Advances of 2020.

I understand the pain my family member goes through every single day, said Anaparti, a postdoctoral fellow in internal medicine, Max Rady College of Medicine, Rady Faculty of Health Sciences. If Im able to prevent at least one person from getting the condition, I will think that my research is successful.

When Anaparti joined El-Gabalawys team, he was tasked with researching the cellular switches that turn genes on and off.

Anaparti studied First Nations people, who have elevated rates of rheumatoid arthritis, to figure out what factors precede or aid in the onset of the chronic disease. One of the factors is cellular switches called microRNAs. Anaparti was interested in people who had levels of anti-citrullinated protein antibody (ACPA), a type of autoantibody whose levels are routinely measured to assess the risk of developing rheumatoid arthritis.

Anaparti found that the first-degree relatives of patients with rheumatoid arthritis have altered levels of certain microRNAs and these individuals exhibit a microRNA expression pattern similar to rheumatoid arthritis patients. Anaparti found that altered microRNA expression in ACPA-positive first-degree relatives is consistent over time and how theyre expressed varies depending on the type of immune cell.

We were able to detect these microRNAs in people years before they developed rheumatoid arthritis, Anaparti said. We have the potential to identify individuals before they become a patient.

Another discovery that Anaparti made was that the methylation, a cellular switch, of specific genes in rheumatoid arthritis patients was different from healthy people and might play a crucial role in the diseases development. He also identified that the levels of some metabolic markers are shifted in at-risk, ACPA-positive first-degree relatives of people with the chronic inflammatory disorder.

Anaparti also found that methylation and microRNAs have a direct influence on the expression of certain genes involved in rheumatoid arthritis.

He has published his findings in reputed journals, including Arthritis & Rheumatology and Arthritis Research & Therapy. His research has been funded by Research Manitoba, Arthritis Society of Canada and Health Sciences Centre Foundation.

Vidyanand is a bright and creative scientist who is meticulous in his experimental approach, El-Gabalawy said. He is an integral part of our human immunology research team and is an excellent mentor for trainees in our lab.

Using data generated from Anapartis research over the last five years, El-Gabalawys team has secured funding from the Canadian Institutes of Health Research for a project focused on rheumatoid arthritis in First Nations people that will include a clinical trial to see whether the combination of turmeric, omega-3 and vitamin D can reduce the risk of rheumatoid arthritis.

Currently in the lab, Anaparti is working on advancing his findings on microRNA by trying to understand their biological role.

The ultimate objective is to prevent people from getting rheumatoid arthritis, Anaparti said. All of this research, all of this understanding, is a step forward in the right direction.

Matthew Kruchak

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UM Today | Rady Faculty of Health Sciences | On the path to preventing rheumatoid arthritis - UM Today

In the UK, more than 10 million people have arthritis or other, similar conditions that affect the joints. Osteoarthritis is the most common type of arthritis in the UK, affecting nearly nine million people. As the NHS explains, osteoarthritis initially affects the smooth cartilage lining of the joint.

A review published in the journal Dovepress aimed to summarise the anti-osteoarthritic effects of curcumin derived from clinical and preclinical studies.

Many clinical trials had been conducted to determine the effectiveness of curcumin in osteoarthritic patients.

According to the review, patients with osteoarthritis showed improvement in pain, physical function, and quality of life after taking curcumin.

To arrive at this conclusion, a total of 15 relevant clinical studies on the effects of curcumin supplementation on osteoarthritic patients were retrieved.

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While some food items are anti-inflammatory, others can make osteoarthritis worse so are best to be avoided.

According to the British Dietetic Association (BDA), Several popular diet books on arthritis advocate avoiding foods such as dairy products or the nightshade vegetables (tomatoes, potatoes, bell peppers and aubergines).

"Though there is some evidence that food avoidance may help rheumatoid arthritis patients, there is no evidence of benefit in osteoarthritis patients," says the BDA.

Diet is not the only remedy for osteoarthritis - exercise can help too.

Although it may seem counterintuitive, research shows that people can and should exercise when they have osteoarthritis.

"In fact, exercise is considered the most effective, non-drug treatment for reducing pain and improving movement in patients with osteoarthritis," explains the Arthritis Foundation (AF).

Different types of exercise play a role in maintaining and improving the ability to move and function.

According to the AF, walking and aquatic exercises are particularly good for most people with osteoarthritis.

As the health body explains, aerobic exercise also reduces fatigue and builds stamina, while helping control weight by increasing the number of calories the body uses.

This is important because excess weight can place pressure on the joints, amplifying symptoms.

Examples of this type of exercise includes walking, jogging, bicycling, swimming or using the elliptical machine.

Other exercises for osteoarthritis include:

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Arthritis treatment: Curcumin improves pain, physical function, and quality of life - Express

Cases of long Covid could put the NHS under pressure for many years to come, an expert has warned.

Danny Altmann, professor of immunology at Imperial College London, said people experiencing long-term symptoms of coronavirus could become a burden on the health service equivalent to arthritis.

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Prof Altmann said arthritis is currently the biggest cause on our NHS time and expenditure but said long Covid risked eclipsing it by taking up even more resources.

He was asked on Times Radio whetherlongCovidwould become less of an issue as fewer people become severely ill from coronavirus, to which he replied: Probably not.

I think for me the rule of thumb at the moment looking at thelongCovidcases is theres no absolute correlation with how severe your initial attack was, Prof. Altmann added.

So theres plenty of people out there who barely know they had it and never had a PCR test or antibody test who are suffering massively still fromlongCovid.

It really worries me, the kind of burden Ive been suggesting is equivalent to our NHS burden of arthritis, which is one of the biggest cause on our NHS time and expenditure.

Its a real thing to worry and think about for the future, for patients and healthcare planning.

Long-Covid patients have complained of persistent fatigue, shortness of breath and problems with memory lasting for months after they were initially infected.

And Prof Altmann said the number of patients reporting symptoms ofCovid-19 weeks after becoming ill could be as high as 20 per cent.

He added that a figure of one in 20 people sufferinglongCovid, reported in October by Kings College London, is lower than what is now being reported.

Many people would have 10-20 per cent as their range if you look at the papers on how many people are still reporting significant symptoms several months afterwards, he continued.

Prof Altmann urged the Government to consider openinglongCovidclinics to dealing with the increased pressure on the NHS.

He recently made the case at a World Health Organisation meeting for mechanisms to be in place for people to be referred to relevant specialists.

The point I was trying to make was really a call to arms for some really good solid research and mechanisms here, because theres no point just having the banner above the clinic if we havent got the doctors to sit in them, he continued.

Prof Altmann warned that there is no absolute correlation between severe illness andlongCovid.

He added: Theres plenty of people out there who barely know they had it and never had a PCR test or antibody test who are suffering massively still fromlongCovid.

Additional reporting byPress Association

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Long Covid could create burden on NHS equivalent to arthritis - iNews

Background:This study aimed to investigate the relationship of serum JNK pathway-associated phosphatase (JKAP) expression with rheumatoid arthritis (RA) risk and clinical features, also to explore the longitudinal change of JKAP during etanercept treatment and its relationship with etanercept treatment response in RA patients.

Methods:A total of 87 RA patients and 44 healthy controls (HCs) were enrolled; then, their JKAP expression in serum was determined by enzyme-linked immunosorbent assay (ELISA). Among 87 RA patients, 42 cases further received the 24-week etanercept treatment; then, their JKAP level in serum (detected by ELISA) and clinical response (evaluated by disease activity score in 28 joints (DAS28) score) were evaluated at week 4 (W4), week 12 (W12), and week 24 (W24) after initiation of etanercept treatment.

Results:JKAP expression was decreased in RA patients compared to HCs, which disclosed a good predictive value for RA risk. JKAP expression was negatively associated with tender joint count, swollen joint count, erythrocyte sedimentation rate, C-reactive protein, and DAS28 in RA patients, respectively. For RA patients who received 24-week etanercept treatment, their clinical response rate was 0.0%, 33.3%, 50.0%, and 69% at W0, W4, W12, and W24, respectively. Importantly, JKAP was gradually increased during etanercept treatment, whose longitudinal elevation positively related to etanercept treatment response in RA patients.

Conclusion:Circulating JKAP links with decreased RA risk and mild disease activity, whose longitudinal elevation positively relates to etanercept treatment response.

Keywords:JNK pathway-associated phosphatase; disease risk; disease severity; etanercept treatment; rheumatoid arthritis.

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JNK pathway-associated phosphatase associates with rheumatoid arthritis risk, disease activity, and its longitudinal elevation relates to etanercept...