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Coronavirus vaccines can have side effects – that typically means they’re working – Anchorage Daily News

December 14th, 2020 1:56 am

The new coronavirus vaccine appears to be extremely effective - blocking serious illness entirely in randomized trials - and it has passed strict safety reviews and won emergency authorization from regulators in five countries so far, including the United States. But news bulletins in the past week provided a reminder that this remains a revolutionary pharmaceutical agent that will be scrutinized in the months ahead as shots go into arms.

Among the unknowns: To what extent does the vaccine prevent infection vs. simply preventing clinical illness?

Can a vaccinated person who becomes infected, but not sick, transmit the virus to someone else? Thats a pivotal factor in forecasting how rapidly the pandemic will be quashed once there is widespread distribution of vaccines.

Another unknown: How long will the protective effect of the vaccine last?

Scientists will also be vigilant for severe allergic reactions. Last week, two health-care workers in the United Kingdom who were among the first batch of people to get the vaccine after it was authorized developed anaphylaxis, a severe allergic response.

Both were known to have a history of severe allergic reactions, and both were treated and recovered. A third person reportedly suffered a rapid heartbeat. British authorities issued new guidance saying people with a history of anaphylaxis should consult with their doctor before taking the vaccine. Researchers do not know what substance in the vaccine formula triggered the severe allergic response.

When you make a decision to launch a vaccine like this, its not because you know everything, said Paul Offit, a pediatrician and vaccine expert at Childrens Hospital of Philadelphia and member of a Food and Drug Administration advisory panel that endorsed the vaccine Thursday. But he added, I think we know enough.

Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, said Saturday, I dont think that the allergic reactions are even close to being a show-stopper for the Pfizer vaccine.

He said the criteria for participation in the random trials excluded people with a history of severe allergic reactions, and it is not surprising that, as the vaccine reached the general population, such rare allergic responses emerged. He said officials will continue to monitor the safety of the vaccines long after they have received emergency authorization.

Observation of safety does not end when you start administering vaccines to the general public, he said.

The big picture is that covid-19, the illness caused by the coronavirus, is a known killer, and has already taken nearly 300,000 lives in the United States and more than 1 million worldwide. Vaccines are essential to crushing the pandemic. U.S. public health officials hope at least 70 percent of the population will agree to be inoculated with one of the vaccines rolled out in the coming months.

I feel like were doing something historic, and theres multiple vaccines, and we should be able to lick this, said immunologist Stanley Perlman of the University of Iowa, who is also a member of the advisory panel that voted Thursday to bless the Pfizer-BioNTech vaccine.

But he acknowledged he is concerned about potential side effects that may not yet have been identified.

I worry about something coming up that we dont know anything about. The unknown, he said.

Two criteria for a good vaccine are effective and safe. The coronavirus vaccine technically named BNT162b2 and developed by industry giant Pfizer and BioNTech meets both standards, according to the professionals who have developed the vaccine, conducted randomized clinical trials and reviewed resulting data during the past several months.

It has received emergency authorization from the FDA and from regulators in the United Kingdom, Canada, Bahrain and Saudi Arabia. Another, similar vaccine frombiotechnology companyModerna is poised to be greenlighted by the FDA this week after a meeting of an advisory panel.

Data from the randomized Pfizer-BioNTech trial showed the two-dose vaccine to be 100 percent effective in preventing severe illness from covid-19.

But in roughly half the people who get the shot, it can produce modest side effects, including fever, headache, fatigue and pain at the injection site. Thats typical for most vaccines.

This is not a flaw or a failure, vaccine experts hasten to point out. Side effects are a sign the immune system is kicking into gear, as intended. Theyre a feature and not a bug, to borrow the language of computer programmers.

Things like fever or soreness at the injection site are normal, and actually they indicate that your body is reacting to the vaccine, which is what you want, said Ellen F. Foxman, an immunologist at the Yale School of Medicine. Thats a good thing.

Side effects were roughly the same in trial volunteers who got the vaccine and those who got a saltwater placebo.

The immune system needs a better public-relations team, because its just the immune system doing what it does, Offit said.

The newly authorized vaccine, like the Moderna shot, uses a synthesized scrap of genetic information, called messenger RNA, that is wrapped in a protective fat layer to keep it from disintegrating. When it goes into cells in the muscle of the upper arm, it incites cellular machinery to manufacture a protein that mimics the shape of the spike protein that protrudes from the surface of the coronavirus.

At no point, in this type of vaccine, is the coronavirus itself or even part of the coronavirus injected into the body. The body, in effect, becomes the vaccine maker, creating a new protein that triggers an immune response. The immune system manufactures antibodies that can disable anything with structural features resembling this protein - including the coronavirus.

Such a vaccine has never been deployed before.

Its very important to think about the whole picture, Foxman said. The vaccine prevents a disease that we know has a lot of bad outcomes, right? Mortality is an outcome - death.

She added, To me its very clear its very beneficial to avoid all the known problems of getting covid 19. I would take this vaccine in a minute if I were offered it.

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Investigating the importance of the Schistosome digestive tract in host immune evasion, parasite survival and novel vaccine development – BugBitten -…

December 14th, 2020 1:56 am

Male and female Schistosomes.

Credit: Alaa. Source: creativecommons.org/licenses/by-sa/4.0>, via Wikimedia Commons https://commons.wikimedia.org/wiki/File:Couple_of_Schistosoma_mansoni.jpg

Schistosomes are a group of parasitic flatworms responsible for causing the neglected tropical diseases of intestinal and urinary schistosomiasis. According to the World Health Organisation, human schistosomiasis is prevalent across 78 countries, in both tropical and sub-tropical regions, and is estimated to affect over 200 million people globally. Schistosomiasis is also considered by the W.H.O. to rank second only to malaria among parasitic diseases in terms of prevalence and socioeconomic burden in an infected community.

The enormous public health and economic burden results in infected individuals and their families being stuck in a cycle of poverty; where they are unable to improve their health due to financial constraints, but also struggle to maintain economic productivity because of their poor health.

In large part, these health, economic and social consequences of schistosome infection are caused by chronic, long-term infections. Without treatment, these parasitic worms have been shown to be able to persist inside a human host for over a decade (here is a case where they had been present for over 38 years!), but how can they survive inside a hosts blood vessels for so many years?

Schistosome physiology enables long-term infection

Schistosomes have a hugely complex life cycle, with an intermediate snail host and a definitive mammalian host.

Upon mammalian host invasion, schistosomes transform through multiple body plans (Cercariae, Schistosomula and Adult), where they eventually reside in their mammalian hosts vasculature, pair with an individual of the opposite sex, and produce thousands of eggs.

However, this is not a happily ever after for our pair of parasitic worms. The hosts vasculature is an extremely hostile environment, brimming with a range of immune components and molecules, tirelessly trying to recognise, bind and kill the parasites. To survive for such a long time inside a human host, schistosomes have (had to) become master manipulators of their environment and display evolutionarily refined characteristics to ensure their long-term survival.

It is well documented that the Schistosome outer surface layers (the tegument) are crucially important in facilitating host immune evasion and maintaining long-term intravascular infection, as this area is highly accessible to immune molecules. However, the parasites digestive tract has now been found to be equally as valuable in helping the parasite to evade the hosts immune system.

The oesophageal gland is essential for survival inside the mammalian host

Jayhun Lee and colleagues at the Morgridge Institute for Research and Howard Hughes Medical Institute of UoW- recently published invaluable research into schistosome digestive tract development, potential mechanisms behind prolonged schistosomiasis infection, and methods of host immune system evasion.

The researchers found, much to their surprise, that an accessory organ of the schistosome digestive tract called the oesophageal gland (OG) develops before the rest of the digestive system. As the digestive system hasnt been fully formed, and blood feeding hasnt started yet, it led Jayhun and colleagues to suggest that the OG has a role in schistosome processes beyond just nutrient uptake and host blood digestion.

For example, the OG is known to secrete a diverse assortment of proteins and other molecules, many of which have unknown functions beyond their demonstrated host protein interaction capabilities. One hypothesis (increasingly being shown to be true) is that OG protein secretions can bind, block, and mop up host antibodies and certain other immune cells before they enter the schistosomes blind gut, acting as a barrier to the hosts immune system. This OG secreted protein barrier could prevent immune molecules from causing direct damage to the gut, or indirect damage by further enhancing the immune response.

In one of their experiments to test the role of the OG in parasite survival, FoxA (a Forkhead-box transcription factor), a key regulator in OG development and maintenance was knocked out using RNAi, resulting in the complete absence of a normal functioning OG.

These parasites lacking an OG were rapidly killed by the hosts immune system in normal immunocompetent mice. However, in mice genetically engineered to lack an immune response, the parasites without an OG survived- showing that, without the OG to mediate protection from the hosts immune molecules, white blood cells could gain access to the parasites gut, enabling recognition of parasite gut tissues, and significantly enhancing killing of the parasite from the inside out.

How can we take advantage of these mechanisms?

Identification of OG-secreted proteins (that manipulate the hosts immune system) and analysis of their interactions with host immune molecules could provide novel antigens with great potential as vaccine targets.

One such group of secreted proteins with considerable promise are the Venom Allergen-Like (VAL) proteins, a group found in a variety of other parasitic worms and organisms. Several VAL proteins have been shown to be released from the OG in Schistosoma mansoni, namely VAL-6, VAL-7 and VAL-13 proteins; however, the specific roles and exact functions of these proteins in the immune evasion process still remains largely unknown.

Additionally, the authors suggest that the identification of genes and proteins downstream of FoxA may help us to selectively disrupt the proper function of the OG, promoting and facilitating schistosome killing by the hosts immune system. Also, as the OG develops during schistosome larval stages, any therapeutic targets could be effective against both immature/larval stages and adult worms, something that the current drug of choice, Praziquantel, cannot do.

With future efforts and continued research into the OG and its associated protein secretions it is possible we may discover a highly promising, novel antigen with great promise as a vaccine target something that will be essential to control and eliminate this disease going forward.

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New COVID-19 Testing Approach That Measures Immune Response Can Be Combined with Standard PCR Tests for Accurate Diagnosis – HospiMedica

December 14th, 2020 1:56 am

A new approach for COVID-19 testing that detects a distinct pattern of immune gene expression in infected individuals could be used as a check against possible errors generated by the standard tests that directly detect the SARS-CoV-2 virus.

Researchers from University of California, San Francisco (UCSF; San Francisco, CA, USA) and Chan Zuckerberg Biohub (San Francisco, CA, USA) have developed the new COVID-19 testing approach that measures a patients immune response for better diagnosis. The new testing approach analyzes completely different molecules - from the person infected, rather than from the virus that infects the person although it can be implemented using the same PCR technology on the same nasal swab samples. It could be used as a standalone test, or even combined into the same testing panels used in standard PCR tests to detect the virus. Combining the technologies could lessen the chances of false negative or false positive results, according to the researchers.

The UCSF scientists created three proof-of-concept versions of the new test - one based on readouts of gene activity from three key genes, one based on readouts from 10 genes, and one based on 27 genes. The tests independently detected COVID-19 infection in clinically confirmed cases, increasing in sensitivity with the number of genes included. The researchers aim to use one of these measures of gene activation both to flag false negative viral PCR tests, in which direct viral detection fails, and to rule out false positive results, which may arise from cross-contamination between samples in testing labs.

To determine which changes in gene activity were distinctive to SARS-CoV-2 infection the researchers first surveyed all the genetic material in swab samples from the upper respiratory tract, so that they could identify the most important and predictive indicators. The researchers examined samples from patients with respiratory symptoms who were tested for COVID-19 as a possible explanation of their illness. The tests showed many of the patients did have COVID-19, but some of them turned out to be infected with more common respiratory viruses (like the flu) or to be suffering from nonviral conditions.

With computer algorithms and a great deal of number crunching, the UCSF scientists were able to identify a distinct pattern of gene expression associated with a tamping down of specific immune responses that occurs early during SARS-CoV-2 infection. The changes differed from those seen in other viral respiratory infections or non-viral respiratory illnesses, allowing for a specific diagnosis of COVID-19. The pattern of immunosuppressive gene expression the researchers identified in COVID-19 may explain the stealthy nature of this highly transmissible virus, according to the researchers.

"Without even having to detect the virus itself, these tests to measure changes in the expression of immune-related genes can determine whether or not someone has COVID-19," said co-senior study author Chaz Langelier, MD, PhD, assistant professor in the Division of Infectious Diseases in the UCSF Department of Medicine.

"We have concluded from our work that there is an immunosuppressive effect taking place that prevents symptoms from developing early during infection despite high levels of viral replication. It's a brilliant strategy, if you're a virus," added Langelier. "Our findings of a diminished inflammatory response by the innate immune system suggest that treatments that suppress the immune system early during COVID-19 infection are unlikely to be beneficial."

Related Links:University of California, San Francisco Chan Zuckerberg Biohub

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We Think ISR Immune System Regulation Holding (STO:ISR) Can Afford To Drive Business Growth – Simply Wall St

December 14th, 2020 1:56 am

We can readily understand why investors are attracted to unprofitable companies. For example, although software-as-a-service business Salesforce.com lost money for years while it grew recurring revenue, if you held shares since 2005, you'd have done very well indeed. But the harsh reality is that very many loss making companies burn through all their cash and go bankrupt.

Given this risk, we thought we'd take a look at whether ISR Immune System Regulation Holding (STO:ISR) shareholders should be worried about its cash burn. In this article, we define cash burn as its annual (negative) free cash flow, which is the amount of money a company spends each year to fund its growth. Let's start with an examination of the business' cash, relative to its cash burn.

Check out our latest analysis for ISR Immune System Regulation Holding

A company's cash runway is the amount of time it would take to burn through its cash reserves at its current cash burn rate. As at June 2020, ISR Immune System Regulation Holding had cash of kr49m and such minimal debt that we can ignore it for the purposes of this analysis. In the last year, its cash burn was kr28m. So it had a cash runway of approximately 21 months from June 2020. That's not too bad, but it's fair to say the end of the cash runway is in sight, unless cash burn reduces drastically. You can see how its cash balance has changed over time in the image below.

ISR Immune System Regulation Holding didn't record any revenue over the last year, indicating that it's an early stage company still developing its business. Nonetheless, we can still examine its cash burn trajectory as part of our assessment of its cash burn situation. Over the last year its cash burn actually increased by 4.9%, which suggests that management are increasing investment in future growth, but not too quickly. However, the company's true cash runway will therefore be shorter than suggested above, if spending continues to increase. ISR Immune System Regulation Holding makes us a little nervous due to its lack of substantial operating revenue. We prefer most of the stocks on this list of stocks that analysts expect to grow.

While its cash burn is only increasing slightly, ISR Immune System Regulation Holding shareholders should still consider the potential need for further cash, down the track. Issuing new shares, or taking on debt, are the most common ways for a listed company to raise more money for its business. One of the main advantages held by publicly listed companies is that they can sell shares to investors to raise cash and fund growth. We can compare a company's cash burn to its market capitalisation to get a sense for how many new shares a company would have to issue to fund one year's operations.

ISR Immune System Regulation Holding's cash burn of kr28m is about 14% of its kr195m market capitalisation. Given that situation, it's fair to say the company wouldn't have much trouble raising more cash for growth, but shareholders would be somewhat diluted.

Even though its increasing cash burn makes us a little nervous, we are compelled to mention that we thought ISR Immune System Regulation Holding's cash runway was relatively promising. While we're the kind of investors who are always a bit concerned about the risks involved with cash burning companies, the metrics we have discussed in this article leave us relatively comfortable about ISR Immune System Regulation Holding's situation. On another note, ISR Immune System Regulation Holding has 4 warning signs (and 2 which make us uncomfortable) we think you should know about.

Of course ISR Immune System Regulation Holding may not be the best stock to buy. So you may wish to see this free collection of companies boasting high return on equity, or this list of stocks that insiders are buying.

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This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. *Interactive Brokers Rated Lowest Cost Broker by StockBrokers.com Annual Online Review 2020

Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team@simplywallst.com.

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Brothers launch clothing line to fund research for a cure to blindness – The Week

December 12th, 2020 8:01 am

Through their Two Blind Brothers clothing line, Bryan and Bradford Manning aim to dress people in stylish yet comfortable clothing while also funding research to cure degenerative eye conditions.

The Manning brothers were both diagnosed with Stargardt disease, a rare genetic eye disorder that can cause blindness, when they were kids. Bryan told People that when you are blind, "the one thing you learn quickly is trust. You have to put these little moments of trust in people, like the trust that a cab driver will drop you off in the right corner, a waiter will give you a good meal recommendation, or you'll get the right change from a cashier because you can't see for yourself."

Bryan and Bradford launched Two Blind Brothers in 2016 so their customers could trust them they sell mystery boxes that are filled with different items, such as hoodies, sunglasses, and socks, which have braille stitched into them. Proceeds benefit organizations like the Foundation for Fighting Blindness, and so far, the brothers have raised more than $750,000.

Two Blind Brothers is more than a clothing line it's also a lifeline. Bryan and Bradford regularly speak with people who were just diagnosed with eye conditions, as well as parents whose children are dealing with eye disorders. "Bryan and I didn't have people around us growing up with this condition," Bradford told People. "The opportunity to make someone with vision impairment or blindness feel better about themselves and live their lives, that drives us." Catherine Garcia

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INDIE VIEW: Seeing through THE IMPENDING BLINDNESS OF BILLIE SCOTT – Comics Beat

December 12th, 2020 8:01 am

The Impending Blindness of Billie ScottBy Zoe ThorogoodAvery Hill Publishing

Is it art without humanity? Thats probably an unanswerable question and certainly, plenty of arguments exist that fall firmly on the side of it doesnt matter or humanity is not a pre-existing necessity for anything, but The Impending Blindness of Billie Scott certainly falls on the side of advocating for a human component to art. Its not likely to change your view, but it will offer an engaging street drama in its attempt, while it posits some big ideas about how art is made and what gives it depth and growth.

Billie Scott is a young woman who lives in shared housing, but keeps to herself, holing up in her room and focusing on honing her art at the cost of human relations and, indeed, being out in the world. Two major events slam her at the same time she has the opportunity for her first gallery show and has to create a body of work in a limited amount of time, but she also finds out that some vision problems shes recently encountered point to impending blindness.

These would seem to be working against each other, but Billie is inspired to seize the moment and takes off on a road trip with the goal of painting 10 portraits of 10 people. This is brought on by an unexpected bonding session with the housemates she previously ignored and the realization that shes been so focused on putting her art first that shes cut herself off from life. Creating the 10 portraits is her opportunity at one last chance of living life while she still has eyesight.

The Impending Blindness of Billie Scott is the debut graphic novel by Zoe Thorogood, who creates a fully-realized street-level world for Billie Scott to inhabit along with a variety of new acquaintances. Thorogood has a scrappy art style and in a strange way, it reminds me of British comics from 30 years ago, the sort of style that might have appeared in 2000 AD, maybe reminiscent of Tank Girl. The subject matter is the exact opposite, but this stylistic connection in my brain added to the tone of the book.

I dont have to tell you and you probably dont have to ask Billies journey is going to be transformative. The beauty of the book doesnt lie in the prospect of there being any other outcome, but in the way that inevitable outcome happens and in the real revelation of the book. Art is not created by playing it safe, and yet that is what Billie has done all her life by living cloistered and focused. She created a world in which it was safe to make her art unimpeded and it was safe to follow her creative track without challenges.

But art is a lot like life, and its by allowing yourself to encounter the unknown that either becomes enriching, partly because thats when you learn new things and find new ways to look at the old things, but also because it can require creative solutions and thats the situation where most people meet failure. Billie Scott has spent her life fearing failure but when confronted by impending blindness she realizes that you eventually encounter the unknown whether you do so intentionally or not. Her 10-portrait project becomes a crash-course in facing the unknown and rising to the challenges it pushes at her.

The irony is that such a venture will also push her art further just as she loses a crucial aspect of creating it. But thats another point in what Billie does shes going to need guile in order to find a new way to express herself. Thats her challenge. But shes also going to require community. The Impending Blindness of Billie Scott shows that the adventure of life and the adventure of art as well as the connections each can build are impossible to separate. And theres no reason anyone should want to.

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WEB EXTRA: A list of illnesses that lead to vision loss or blindness, tips to prevent them – KLAS – 8 News Now

December 12th, 2020 8:01 am

LAS VEGAS (KLAS) Many think blindness is an illness that people are either born with or a condition that a person experiences after an accident.

8 News Now spoke with Dr. Brian Alder, an Ophthalmologist and Corneal Specialist at Shepherd Eye Center to explain and list the illnesses that can lead to blindness. Dr. Alder says some of the illnesses that lead to blindness are developed over time and completely preventable.

EYE ILLNESSES THAT CAN LEAD TO BLINDNESS

TIPS TO PREVENTING EYE ILLNESSES:

COMMON EYE ILLNESSES AND TREATMENTS:

Cataracts: Dr. Alder says that while cataract patients in the US have access to treatment that can address this issue, in other countries it is one of the leading causes of blindness. Dr. Alder says cataract surgery can easily fix the condition that causes his patients to lose significant vision.

Macular Degeneration: The center of the patients retina deteriorates. Dr. Alder says there are steps that can be taken to mitigate the loss of vision if you have this condition.

Glaucoma: A condition that damages the optic nerve which is vital for good vision, can be treated. Dr. Alder says an eye exam can help to uncover this condition and with proper treatment, patients can avoid the complications of severe vision loss or blindness.

Dr. Alder says some glaucoma patients have no symptoms so he recommends that even if your eyes seem fine to schedule an eye exam. An eye exam can reveal high eye pressure that will eventually cause slow vision loss. With Glaucoma, Dr. Alder says you can have no blurriness in your central vision but have significant vision loss in your peripheral vision until you experience tunnel vision.

Dr. Alder says these three common eye conditions all develop over time and with regular eye checks and treatment all can be avoided to preserve your vision.

In addition to getting an exam and following the treatment plan prescribed by your doctor, you can take care of your eyes by:

For more information please visit the website Academy of Ophthalmology at http://www.aao.org.

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Hyderabad Doc Who Returned From US to Help the Blind Wins $3 Million Global Prize – The Better India

December 12th, 2020 8:01 am

The LV Prasad Eye Institute was chosen for the Greenberg Prize End Blindness 2020, and the Outstanding Achievement award will be presented to its founder, Dr Gullapalli Nageswara Rao.

LV Prasad Eye Institute (LVPEI), a World Health Organisation (WHO) collaborating centre for the prevention of blindness, is a comprehensive eye health facility headquartered in Hyderabad, Andhra Pradesh. Recently, it was chosen for the Greenberg Prize End Blindness 2020 and the Outstanding Achievement award, which will be presented to its founder Dr Gullapalli Nageswara Rao.

The purpose of the Greenberg Prize is to create a worldwide research community that will contribute its collective skills and resources to end blindness. The winners are chosen based on the strength of their contributions towards this goal and are awarded prize money of $3 million.

LVPEI, with 3 tertiary, 20 secondary and 184 primary care centres, has benefitted more than 15 million patients in India. Here are some things you should know about Dr Rao:

Dr Rao decided to become an Ophthalmologist because he wanted to follow the footsteps of his father, who was a legendary doctor named Govindappa Venkataswamy and the founder of Aravind Eye Hospital, Chennai.

After completing his basic medical education in Guntur, Andhra Pradesh, Dr Rao completed his postgraduate residency training in Ophthalmology at the All India Institute for Medical Sciences (AIIMS), New Delhi. In 1974, he went to the United States of America where he trained at the Tufts University School of Medicine in Boston. Then, he trained and taught for a while at the Rochester School of Medicine.

Apart from training abroad, Dr Rao was also a visiting professor at several universities in the USA, Europe, Australia and Asia. His areas of specialisation include diseases of the cornea, eye banking, corneal transplantation, eye care policy and planning.

Till date, Dr Rao has published more than 300 papers in national and international journals and has served on the editorial boards of several journals.

In 1981, Dr Rao and his wife returned to India. In an interview with Factor Daily, he says that he decided to return to India because he wanted to build an eye centre that would encompass patient care, education, and research, in Hyderabad.

After he returned, he donated all his savings to ophthalmic corporations, and he approached the former Chief Minister of Andhra Pradesh NT Rama Rao to provide him land to establish an educational institute. The land allotted by the CM was used to open the public health and optometric education department.

In 1985, he further received Rs 5 crores and 5 acres of land from Ramesh Prasad, the son of popular movie director LV Prasad after which he launched the LV Prasad Eye Institute.

The former secretary-general and later the CEO of the International Agency for Prevention of Blindness (IAPB) played a pivotal role in developing and fostering the global initiative to eliminate avoidable blindness along with WHO.

In 2002, he was honoured by the Government of India with the fourth highest Indian civilian award Padma Shri.

In 2017, Dr Rao was inducted into the Ophthalmology Hall of Fame at the meeting of American Society of Cataract and Refractive Surgery (ASCRS) in Los Angeles. In the past three centuries, only 57 ophthalmologists from around the world have been inducted into the Hall of Fame.

During an interview with Forbes magazine, Dr Rao shares that when KR Narayanan, the former President of India, developed a cataract, his secretary, Gopalkrishna Gandhi, asked for his opinion. Though the first citizen of India was recommended surgery, Dr Rao did not do it himself because he had stopped operating after he turned 55. When the Presidents secretary asked him to do them a favour and conduct the operation himself, Dr Rao reportedly replied with I am doing you a favour by not operating on him.

On being awarded the Greenberg Prize, Dr Rao said, I feel humbled and honoured to accept this prestigious award on behalf of the 3000-strong family of LVPEI. Eliminating avoidable blindness by the year 2020 has been an aspiration of the global eye care community for over two decades.

According to news reports, the Greenberg Prize awards ceremony will be live-streamed at 5:30 IST on 15 December 2020, and the link can be found on the official website.

(Edited by Yoshita Rao)

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Researchers Say We’re Watching The World Go Blind – Newswise

December 12th, 2020 8:01 am

Newswise As the global population grows and ages, so does their need for eye care. But according to two new studies published inThe Lancet Global Health, these needs arent being met relative to international targets to reduce avoidable vision loss.

As 2020 comes to a close, an international group of researchers set out toprovide updated estimates on the number of people that are blind or visually impaired across the globe, to identify the predominant causes, and to illustrate epidemiological trends over the last 30 years.

This is important because when we think about setting a public health agenda, knowing the prevalence of an impairment, what causes it, and where in the world its most common informs the actions that key decision makers like the WHO and ministries of health take to allocate limited resources, saysJoshua Ehrlich, M.D., M.P.H., a study author and ophthalmologist atKellogg Eye Center.

The study team assesses a collection of secondary data every five years, undertaking a meta-analysis of population-based surveys of eye disease assembled by the Vision Loss Expert Group and spanning from 1980 to 2018.

A study like this poses challenges since regional populations vary in age.

For example, the population in some Asian and European countries is much older on average than the population in many African nations. Many populations are also growing older over time. A direct comparison of the percentage of the population with blindness or vision impairment wouldnt paint a complete picture says Ehrlich, who is also a member ofUniversity of Michigans Institute for Healthcare Policy and Innovation, explains.

To address this issue, the study looked at age-standardized prevalence, accomplished by adjusting regional populations to fit a standard age structure.

We found that the age-standardized prevalence is decreasing around the world, which tells us eye care systems and quality of care are getting better, says study authorMonte A. Del Monte, M.D., a pediatric ophthalmologist at Kellogg Eye Center. However, as populations age, a larger number of people are being affected by serious vision impairment, suggesting we need to improve accessibility to care and further develop human resources to provide care.

In fact, the researchers found that there wasnt any significant reduction in the number of people with treatable vision loss in the last ten years, which paled in comparison to the World Health Assembly Global Action Plan target of a 25% global reduction of avoidable vision loss in this same time frame.

Although findings varied by region globally,cataracts and the unmet need for glasses were the most prevalent causes of moderate to severe vision impairment.Approximately 45% of the 33.6 million cases of global blindness were caused by cataracts, which can be treated with surgery.

Refractive error,which causes a blurred image resulting from an abnormal shape of the cornea and lens not bending light correctly, accounted for vision loss in 86 million people across the globe. This largest contributor to moderate or severely impaired vision can be easily treated with glasses.

Also important, vision impairment due to diabetic retinopathy, a complication of diabetes that affects eyesight, was found to have increased in global prevalence.

This is another condition in which we can prevent vision loss with early screenings and intervention, says study authorAlan L. Robin, M.D., a collaborating ophthalmologist at Kellogg Eye Center and professor at Johns Hopkins Medicine. As diabetes becomes more common across the globe, this condition may begin to affect younger populations, as well.

Working as a global eye care community, we need to now look at the next 30 years, Ehrlich says. We hope to take these findings and create implementable strategies with our global partners through ourKellogg Eye Center for International Ophthalmologyso fewer people go blind unnecessarily.

In an effort to contribute to the WHO initiativeVISION 2020: The Right to Sight, the researchers updated estimates of the global burden of vision loss and provided predictions for what the year 2050 may look like.

They found thatthe majority of the 43.9 million people blind globally are women. Women also make up the majority of the 295 million people who have moderate to severe vision loss, the 163 million who have mild vision loss and the 510 million who have visual impairments related to the unmet need for glasses, specifically poor near vision.

By 2050, Ehrlich, Del Monte, and Robin predict 61 million people will be blind, 474 million will have moderate and severe vision loss, 360 million will have mild vision loss and 866 million will have visual impairments related to farsightedness.

Eliminating preventable blindness globally isnt keeping pace with the global populations needs, Ehrlich says. We face enormous challenges in treating and preventing vision impairment as the global population grows and ages, but Im optimistic of a future where we will succeed because of the measures we take now to make a difference.

Both studies were funded by Brien Holden Vision Institute, Fondation Tha, Fred Hollows Foundation, Bill & Melinda Gates Foundation, Lions Clubs International Foundation, Sightsavers International and the University of Heidelberg.

Papers Cited:Causes of blindness and vision impairment in 2020 and trends over 30 years, and prevalence of avoidable blindness in relation to VISION 2020: the Right to Sight: an analysis for the Global Burden of Disease Study,The Lancet Global Health. DOI:10.1016/S2214-109X(20)30489-7

Trends in prevalence of blindness and distance and near vision impairment over 30 years: an analysis for the Global Burden of Disease Study,The Lancet Global Health. DOI:10.1016/S2214-109X(20)30425-3

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Tommy Lister Jr. Was Born Blind in One Eye, but He Didn’t Let That Slow Him Down – Distractify

December 12th, 2020 8:01 am

Before he became well-known in wrestling circles, Tommy grew up in Compton, California, where he developed an interest in films and religion. Initially, it seemed like Tommy might pursue a career devoted to athletics.

He was the NCAA Division II National shot put champion in 1982, and he also tried out for the United States Football League. When he was cut after two games in the league, he decided to pursue acting instead.

Tommy ultimately had a fairly successful career in Hollywood, making guest appearances in a number of TV projects and also earning roles in films like The Dark Knight and Quentin Tarantino's Jackie Brown. His most famous part was in the 1995 film Friday and the movie's sequel five years later.

He also had a voice role in the film Zootopia and most recently appeared in a number of films that were released in 2017.

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Blindness cure on horizon after vision loss fully restored in mice with glaucoma – Study Finds

December 12th, 2020 8:01 am

BOSTON, Mass. A cure for blindness could be on the horizon after a team from Harvard Medical School reports theyve successfully restored vision loss due to glaucoma in mice.

Scientists achieved the feat by turning back the clocks of eye cells using a cocktail of proteins. It is the first time complex tissue has been reprogrammed to an earlier age. Clinical trials are set to start within the next two years. The groundbreaking technique is expected to work just as well in humans and may also conquer other neurological diseases, including dementia.

Researchers add this new study sheds fresh light on the mechanisms behind growing old; pointing to a therapeutic target for a host of conditions.

Our study demonstrates that its possible to safely reverse the age of complex tissues such as the retina and restore its youthful biological function, senior author Professor David Sinclair says in a university release.

According to the Centers for Disease Control and Prevention, over four million people over 40 are legally blind or living with low eyesight in the United States. The team at Harvard made use of a harmless virus to deliver three genes into the retinas of lab rodents with glaucoma the most common cause of human blindness.

Called Oct4, Sox2, and Klf4, these proteins are transcription factors that are switched on during embryonic development. Researchers say the procedure also worked similarly well in elderly mice with diminishing sight due to normal aging.

Afterwards, gene expression patterns and electrical signals of the cells returned to a similar state as in young mice, including improved vision. Study authors explain that their technique could actually heal the damaged optic nerves in the mice with glaucoma.

Although they reside in the eyes and technically outside the skull, the retinal ganglion cells (RGCs) are brain neurons. The team believes their approach can work in restoring other organs too.

If affirmed through further studies, these findings could be transformative for the care of age-related vision diseases like glaucoma and to the fields of biology and medical therapeutics for disease at large, Prof. Sinclair explains.

The study focuses on the epigenetic clock, the aging equivalent of the body clock. It tells genes to switch on or off. It is believed changes to it, either through our DNA or the environment, cause cells to malfunction and trigger age-related diseases.

One of the most important gene processes is methylation. DNA methylation can prevent certain genes from expressing themselves. This, for example, may stop tumor-causing genes from turning themselves on and causing diseases like cancer.

At the same time, genes that should be switched on get turned off and vice versa, resulting in impaired cell function. Over time, methylation also causes DNA to lose their more youthful patterns.

Past work achieved the feat in cells grown in laboratory dishes, but failed to demonstrate the effect in living organisms. Prof. Sinclair and his team targeted cells in the central nervous system as its the first place in the body affected by aging. After birth, its ability to regenerate declines rapidly. The results reveal that treatment doubled the number of surviving cells after optic nerve injury and increased regrowth five times.

At the beginning of this project, many of our colleagues said our approach would fail or would be too dangerous to ever be used, study author Dr. Yuancheng Lu explains. Our results suggest this method is safe and could potentially revolutionize the treatment of the eye and many other organs affected by aging.

In mice with glaucoma, it boosted nerve cell electrical activity and sharpened sight. They could see moving vertical lines on a screen better, even after vision loss had already occurred.

Regaining visual function after the injury occurred has rarely been demonstrated by scientists, co-author Prof. Bruce Ksander adds. This new approach, which successfully reverses multiple causes of vision loss in mice without the need for a retinal transplant, represents a new treatment modality in regenerative medicine.

The treatment worked just as well in 12 month-old mice with diminishing vision due to normal aging. These mice are the equivalent of a person in their 60s. An analysis of molecular changes in treated cells identified reversed patterns of DNA methylation, suggesting it is a driving factor in aging.

What this tells us is the clock doesnt just represent timeit is time, Sinclair concludes. If you wind the hands of the clock back, time also goes backward.

The team describes the findings so far as encouraging. Full body treatment of the mice with the three-gene procedure has produced no negative side effects after a year of testing.

The results appear in the journal Nature.

SWNS writer Mark Waghorn contributed to this report.

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How This Woman Is Using Her Cellphone to Tackle River Blindness in Cameroon – Global Citizen

December 12th, 2020 8:01 am

Why Global Citizens Should Care

On a typical work day in Yaound, Cameroon, Kareen Atekem hops on the back of a motorbike outfitted in a rain jacket, helmet, and face mask. With a phone in hand and a digital map guiding her way, Atekem directs a motorbike driver to her destination.

Soon, the dirt path becomes obstructed by bushes, forcing her to continue the journey by foot. Atekem, a health researcher, ventures quite literally off the beaten path, navigating unmapped areas of the countrys West Region.

Here, she is looking to find members of the nomadic Massangam community small groups of the wider Massangam people, whose geographic locations are not recorded. Atekem uses satellite technology to find clusters of huts, which she presumes to be nomadic camps.

Its not really easy because these camps are far from the community and very hard to reach, Atekem told Global Citizen, breathless, as she walked, swatting bushes to clear her way.

Hours later, she walks on wooden planks that allow her to cross the Nja river, a sign that she is on the right path. When she arrives at a site, Atekem meets members of a settled community not the nomadic group she was seeking. She follows the next location on the map and heads off once again, eventually locating a nomadic community.

It was worth the drive, Atekem said.

Now, her real work begins.

Kareen Atekem is photographed at the Sightsavers headquarters in the Bastos district of Yaound, Cameroon in November 2020. "Since my early childhood I have always wanted to help, to bring my help to people in need," said Atekem.Image: Daniel Beloumou for Global Citizen

The 30-year-old health advocate is leading a research project that uses alternative treatments to accelerate the elimination of onchocerciasis, a neglected tropical disease (NTD) also known as river blindness.

Atekem spends the day at the nomad camp, screening individuals for river blindness, and providing treatment to those who have it.

There has been ongoing transmission of the disease among the Massangam for more than 20 years, despite members with river blindness receiving medication annually, according to Atekem.

She says communities have had prevalence rates of 30%.

This is really, really high, and of course with this prevalence, you cant achieve elimination, she said.

Atekem, who works for the international charity Sightsavers, says their strategy to eliminate river blindness includes providing the treatment mectizan on a biannual basis, instead of an annual basis, in addition to doxycycline, in an effort to accelerate the elimination of the disease. The organization also chemically treats the rivers where the flies breed to kill larvae, preventing them from transforming into adult flies which transmit the disease.

Kareen Atekem takes notes at the Sightsavers headquarters in Yaound, Cameroon. "Since the beginning of the COVID-19 pandemic, we have been doing a lot of teleworking... and the staff is reduced here at the NGO's headquarters," explained Atekem.Image: Daniel Beloumou for Global Citizen

River blindness, an eye and skin disease, is spread by bites from infected black flies near fast-flowing rivers. When flies bite, their worm larvae invade a persons body, causing severe skin irritations. The infection can eventually cause blindness.

Together with her team, Atekem identifies community members from the Massangam nomadic camps to receive training on river blindness.

I need to get to these people to make sure they are actually involved in the treatment and we combat NTDs, Atekem said. If we leave this population out, it would be like you are wasting your efforts [in eliminating the disease].

The trained community members act as liaisons between the researchers and their nomadic communities, helping Atekem seek approval from the camp heads to conduct this work an essential step and translating during camp visits.

Each camp consists of huts, or households, that are typically related, and are groups of 10 to 50 people, within the larger Massangam nomadic population comprising around 700 people, and a settled population of around 2,000.

When we visit normally at nightfall, we camp with them At times we even sleep in their huts and get up in the morning and continue to the next camp, she explained. Once we are finished with one community,we come back to our base and prepare to go to the next one.

Atekem who makes this trip three times a year spends up to a month with the Massangam people. The journey from Cameroons capital, where Atekem lives, involves a five-hour drive to Bafoussam, the West Region capital. From there, its another three-hour drive on unpaved roads to a town, also called Massangam, which acts as base, and an additional three-hour trek by motorbike and foot to reach specific camps.

Growing up in the Northwest Region of Cameroon, Atekem always heard about the Massangam nomads and over three years of working with them, she has an in-depth knowledge about the groups travel patterns, culture, and values.

Kareen AtekemKareen Atekem, Sightsavers' research coordinator, and Souleman Zidane, a community drug distributor, write up information during screening for river blindness at Moussa Camp, Makouopsap community, West Region, Cameroon in 2020. Amidou Sale/Sightsavers

Kareen AtekemSightsavers' research coordinator Kareen Atekem speaks to Bororo women in Makouopsap, WestRegion, Cameroon. The women are waiting to be tested for river blindness in 2018. Dominique Catton/Sightsavers

Sometimes they give you food and water without you asking. They are so hospitable, she said. You have to share with them. I even have one picture where we were eating with them in their traditional bowls.

A few months ago, when Aekem didnt show up as expected, members in the community wondered why.

Laolo, a 19-year-old Massangam man, traveled several hours to access a phone.

He called me and said, Madam,are you not coming to give the treatment? I said, Because of the coronavirus, we have halted all our activity. He said, What is the coronavirus? Atekem recalled.

Atekem informed him about the pandemic and shared how his community could take precautions.

Related Stories Jan. 8, 2020 How Health Workers Are Reaching a Nomadic Tribe in Sub-Saharan Africa

It was something so impactful to me that I developed a relationship with the nomads that they would call me when Im not around, she said.

Although barriers,including their remote location, culture, and language, pose a challenge in accessing medical services, Atekem says it is essential to reach nomadic populations with health care.

These people love their health and they are willing to do whatever it takes to protect their health, but I fear they are not being reached, she said.

Providing equitable medical care means ensuring no one is left behind in the fight against this disease, she said. Everyone has a right to treatment, not just those who are settled nomads, too.

Kareen Atekem, research coordinator at Sightsavers, stands outside the Sightsavers offices in Yaound, Cameroon in November 2020.Image: Daniel Beloumou for Global Citizen

The Last Milersis a profile series that highlights the people tackling neglected tropical diseases (NTDs),which impact more than 1 billion people globally.By working to ensure equitable access to preventative measures, treatments, and information, these people support the elimination of NTDs in various ways, across different fields. These advocatesaim to reachevery last milewith necessary health care tools and services.

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Organoids to rebuild the brain | Penn Today – Penn Today

December 12th, 2020 8:01 am

Computer-augmented brains, cures to blindness, and rebuilding the brain after injury all sound like science fiction. In recent years, these advances are closer to reality than some might realize, and they have the ability to revolutionize neurological care.

Neurologic disease is now the worlds leading cause of disability, and upwards of 11 million people have some form of permanent neurological problem from traumatic brain injuries and stroke.

Thanks to recent advances, sometimes lasting neurologic disease can be prevented. If a stroke patient is seen quickly enough, life-threatening or -altering damage can be avoided, but its not always possible. Current treatments to most neurologic disease are fairly limited, as most therapies, including medications, aim to improve symptoms but cant completely recover lost brain function.

H. Isaac Chen, an assistant professor of neurosurgery at the Perelman School of Medicine and a neurosurgeon at the Corporal Michael J. Crescenz Veterans Affairs Medical Center, is working to address this challenge. Chen calls the effort to improve how people function neurologicallyinstead of addressing disease symptomsthe holy grail of clinical neuroscience.

This quest drives my entire academic careerbeing able to treat patients who dont really have other options right now by repairing the brain, Chen says. While there are efforts to try to prevent disease and damage, there will always be patients who end up with permanent neurological problems.

Chen suspects that implanting neural tissue like a brain organoid could rebuild brain circuitry. His research is focused on the cerebral cortexthe part of the human brain that sets us apart from other animals. The cerebral cortex supports basic functions such as movement, visual sensation, and higher-order cognitive processes, like working memory and the ability to plan.

This story is part one of a series, Science Fiction Meets Neuro-Reality. Read more at Penn Medicine News.

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Vision Care Gene Therapy Acquired | INVISIONMAG.COM – InvisionMag

December 12th, 2020 8:01 am

RARITAN, NJ Janssen Pharmaceuticals Inc. has acquired rights to an investigational gene therapy from Hemera Biosciences LLC.

The therapy, called HMR59, is administered as a one-time, outpatient intravitreal injection to help preserve vision in patients with geographic atrophy, a late-stage and severe form of age-related macular degeneration.

Financial terms of the deal were not disclosed.

Patients with AMD often have low levels of CD59, a protein that protects the retina from damage caused by an essential part of the bodys natural immune response called complement, according to a Janssen press release. In geographic atrophy, an overactivity of complement destroys cells in the macula, the central part of the retina responsible for central vision and seeing fine details, and results in a relentless progression to blindness. HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, helping to prevent further damage to the retina and preserve vision.

Geographic atrophy affects 5 million people globally, and is a leading cause of blindness in people over 50 years of age. The prevalence of geographic atrophy increases as the global population ages with roughly one in 29 people over age 75 affected, and nearly one in four people over age 90. There are currently no available therapies other than vitamins and low vision aids, Janssen notes.

Geographic atrophy is a devastating form of AMD that impacts the ability to accomplish everyday tasks, such as reading, driving, cooking, or even seeing faces, said James F. List, MD, PhD, global therapeutic area head, cardiovascular and metabolism, Janssen Research & Development LLC. Our aim with this novel, single-administration gene therapy is to use our development expertise and deep heritage in vision care to help improve patient outcomes by intervening early, halting the progression to blindness, and preserving more years of sight.

ThePhase 1 studyof HMR59 for patients with geographic atrophy is complete. A second Phase 1 study exploring HMR59 in patients with wet-AMD is currently conducting follow-up visits to evaluate long-term safety.

Janssen Pharmaceuticals is one of the Janssen Pharmaceutical Cos. of Johnson & Johnson.

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Mobile : they gave an injection in one eye and the other replicated the genetics – Explica

December 12th, 2020 8:01 am

American scientists were able to cure the blindness in a group of 37 people who suffered from this hereditary condition. The most amazing thing about the study is that they gave an injection in only one eye. Then the other replicated genetics in order to correct the condition. In the substance they combined a gene therapy vector with DNA modified.

Daily Mail review that the study was conducted by scientists from the University of Pittsburgh. In addition, due to their positive results, they achieved a publication in the magazine Science Translational Medicine. There were 37 patients who showed improvement in symptoms of blindness. That is, 78% of those treated, a high enough percentage to consider the study a success.

They explain that modified DNA contains rAAV2 / 2-ND4A substance designed to treat vision loss problems. They noted that the injection applied is capable of migrating from one eye to another. And so the deoxyribonucleic acid improves for both.

DNA prick replaces a mutation that causes blindness

Scientifically details the Dr. Jos-Alain Sahel, from the house of studies mentioned that DNA replaces a mutation found in people who suffer from the type of blindness. Such substitution does the job of removing harmful ones and at the same time installing healthy ones.

What the injection destroys is something called ganglion cells, which are found in the retina. This is what leads to the degeneration of the optic nerve and the acceleration of a worse functioning of the eyes.

Our study offers great hope for the treatment of this blinding disease in adults who are still young, Sahel said.

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SIFI appoints Jelle Kleijn as Global Head of Acanthamoeba Keratitis to globally lead the effort to deliver the first registered treatment for this…

December 12th, 2020 8:00 am

A global pharma executive with extensive experience stemming from drug development, through medical, market access to sales & marketing, Jelle has long standing experience in leadership roles in large pharmaceutical and biotech companies. He and his team have been directly responsible for delivering new treatments to patients suffering from rare and often acutely life-threatening diseases. Jelle holds a PhD and a MSc in Medical Pharmaceutical Sciences from the University of Groningen.

"I have spent many years of my career working actively to find innovative treatment approaches for patients affected by rare and life-changing disorders. It is with great pleasure that I join the SIFI team to help advance treatment for AK, a devastating acute eye infection. No patients should feel left behind as my mission is to make sure we help find everyone with this severe eye infection, and being able to provide them with anapproved treatment, as early as possible," said Jelle Kleijn PhD.

"We are pleased to welcome Jelle Kleijn to the SIFI team," comments Fabrizio Chines, Chairman and CEO, who continues: "Acanthamoeba keratitis is an ultra-rare acute infection of the eye that potentially leads to blindness, and eye loss. Jelle's experience matches the unique challenges that our organisation faces to bring SIFI's first orphan drug to market and, more importantly, the first registered treatment for this severe eye infection. I'm confident that the appointment of Jelle will lead to a positive impact on Acanthamoeba keratitis patients around the world."

SIFI has recently completed patient enrolment in the pivotal phase 3 ODAK trial, comparing polihexanide 0.08% monotherapy versus the combination of polihexanide 0.02% and propamidine 0.1%. SIFI expects top-line results in the second half 2021. Polihexanide has already been granted orphan drug designation for the treatment of AK in both the European Union and United States. It has taken 13 years for the development process of polihexanide as a high-dose 0.08% monotherapy to reach this point. Notably, if approved, it will become the first medicine to be licensed for AK globally.

About polihexanidePolihexanide is an investigational disinfectant, a polymer, in development for the treatment of AK. It acts on both the trophozoites and cysts of the protozoan Acanthamoeba. It is locally administered as a high-dose 0,08% monotherapy, unlike current unlicensed alternatives which usually involve combination therapy with multiple eye-drop medications.

About Acanthamoeba Keratitis (AK)AK is a severe corneal infection caused by the parasite Acanthamoeba. AK is a devastating acute eye infection presenting with unbearable pain and extreme light sensitivity.Each day of treatment delay increases the risk of blindness and eye loss. This means that appropriate and timely management is essential. AK is an ultra-rare condition affecting one to four per million people per year, but its incidence has been growing rapidly in recent years. No treatment is currently licensed for this acute eye infection in any country.

About SIFI SIFI is a leading ophthalmic company, headquartered in Italy, focusing on eye care since 1935. SIFI develops, manufactures, and markets innovative therapeutic solutions for patients with ophthalmic conditions. SIFI is fully committed through its R&D to improve the quality of life of patients, exporting treatments to more than 20 countries worldwide with a direct presence inItaly,Spain,France,Romania,MexicoandTurkey.

Key ContactSabrina Zappia- Sifi Press & Communication Officepress@sifigroup.com+393336999669

Photo - https://mma.prnewswire.com/media/1359441/SIFI_Jelle_Kleijn.jpg

SOURCE SIFI Press & Communication Office

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Gene Therapy in One Eye Improves Vision in Both Eyes – The Scientist

December 12th, 2020 8:00 am

I

n a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.

The paper has very strong clinical implications that a single injection maybe is enough for bilateral effects, says Thomas Corydon, who studies ocular gene therapy at Aarhaus University in Denmark and was not involved in the work.

The onset of Leber hereditary optic neuropathy (LHON) is sudden. Patientsusually young menstart losing vision at the center of one eye. Within months, the other eye follows, leaving them legally blind. The disease is caused by a point mutation in the mitochondrial genome that leads to dysfunction and death of retinal ganglion cells, the axons of which make up the optic nerve. About 70 percent of patients have the same mutation, known as MT-ND4.

If you're going to start somewhere, it makes sense to tackle this variant, says Patrick Yu-Wai-Man, an ophthalmologist at the University of Cambridge in the United Kingdom. He and his collaborators, including teams from GenSight Biologics and Stealth Biotherapeutics and a group led by University of Pittsburg Medical Center ophthalmologist Jos-Alain Sahel, as well as other groups, previously showed that the point mutation could be corrected in animal models and in cell culture using gene therapy.

Its difficult to get genetic material into the mitochondrial genome because mitochondria have two membranes, an outer and inner membrane, Yu-Wai-Man explains. In the clinical trial, he, Sahel, and colleagues overcame this hurdle by injecting an AAV vector containing a wildtype copy of the ND4gene with an added mitochondrial-targeting sequencea strategy that had already been shown to correctly direct the protein product of ND4 and other mitochondrial genes to the organelle.

Each of 37 patients received the therapeutic virus via a single injection into the fluid within one eye six to 12 months after the onset of vision loss. They also got a sham treatment in the other eye: a surgeon pressed the eye with a blunt cannula to simulate an injection.

We thought that, if there was going to be an effect, it would be isolated to that eye and then the other one would be the perfect internal control, Yu-Wai-Man tells The Scientist. But as it turns out, that wasnt the case.

With a slight delay in the sham-treated eye, both eyes started to improve. By 96 weeks after treatment, 29 of the patients had gained visual acuity in both eyes and reported increases in their quality of life.

Patients do improve, but, even with the treatment, they still function at a very low level, says Byron Lam, an ophthalmologist at the University of Miami who was not involved in the study. Most of the subjects were still near legal blindness at the end of the study.

To determine how the bilateral effect might be happening, Yu-Wai-Man and colleagues injected the therapeutic virus into one eye of three monkeys. Three months later, they found viral DNA in the noninjected eye and optic nerve. This raises the possibility that the viral vector supplies the wildtype protein in the untreated eye, but its not firm proof.

Finding viral DNA in the untreated eye in primates is a little short of being definitive because DNA expression alone doesnt prove that youre getting a therapeutic effect. Detecting DNA doesnt mean there is mRNA expression or protein production, says Mark Pennesi, an ophthalmologist at Oregon Health & Science University who did not participate in the work.

Previous work has shown that there could be transneuronal spread of the vector, but we also need to keep a critical mind and think that there might be other explanations, agrees Yu-Wai-Man. It could be that injecting the vector in one eye leads to some form of localized inflammation that induces mitochondrial biogenesis, thus making the mitochondria work better, he adds. Another option is that improvement in one eye leads to reorganization in the part of the brain that interprets signals from the eye, which could enhance vision overall.

Clearly, further investigations are needed to understand the underlying mechanisms of how the interocular diffusion of viral DNA vector occurs and whether there are other mechanisms by which the optic nerves directly communicate, Bin Li, an ophthalmologist at Tongji Hospital in China who was not involved in the study, writes in an email to The Scientist.Li explains that his group has also reported that material injected in one eye can reach the other optic nerve.

These findings have implications for how this type of research should be performed in the future, he writes. Theyve shown that contralateral sham-treated eyes cannot serve as true internal control for clinical studies.

When you read this paper, you get a little excited, and then in some ways, you get a little disappointed, because it does look like theres some kind of positive effect with this treatmentthat it does do something more than what would happen with just the natural history of the disease. Unfortunately, the results are confounded by the fact that you treat one eye, but then there is improvement in the untreated control eye, Pennisi tells The Scientist. The question then really becomes . . . why did you get that result?

Along with academic collaborators, Yu-Wai-Man, who consults for GenSight Biologics and Stealth Biotherapeutics, will continue to explore this question as they focus on ongoing clinical trials of this therapeutic.

P. Yu-Wai-Man et al., Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy,Science Translational Medicine,doi:10.1126/scitranslmed.aaz7423, 2020.

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Protecting children’s vision with early eye care Zambia Daily Mail – Zambia Daily Mail

December 12th, 2020 8:00 am

MELODY MUPETA, KitweGOOD eye care in children is key in early detection of eye conditions such as cataracts, ptosis and squints.These are some of the common eye conditions in children which, if left unchecked, could cause blindness and social and psychological disturbance in a child.According to medical practitioners, paediatric cataracts are responsible for over 3.0 percent of all blindness and they are the second to adult cataract.Ptosis and squints can cause poor vision by reducing visual stimulation in the affected eye, thus preventing the brain from developing good vision in the particular eye.Squints can also be associated with other conditions of the eye like cataracts and tumours if they are left untreated.Lack of information about the three conditions makes people believe the eye conditions in question are incurable.Kitwe Teaching Eye Hospital (KTEH) paediatric ophthalmologist, Chileshe Mboni, says the three eye conditions are complex but manageable.Dr Mboni says cataract is a condition in which the lens of an eye becomes white, while in CLICK TO READ MORE

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I have got your back! – The Tribune India

December 12th, 2020 8:00 am

While environmentalist Balbir Singh Seechewal hasnt visited the Delhi stirs yet, he has been active in the organising support for the activities regarding the same in the region. He dispatched two different jathas of sportspersons, who planned to show solidarity with the farmers protests this week. While he sent off 20 of Punjab sportspersons who visited Delhi to return their awards, and join the stir, he also saw off kabaddi players from Sultanpur Lodhi going to offer support to the farmers. While villagers of Dona at Sultanpur Lodhi have been holding langar for farmers at Delhi, Seechewal has been seeing off jathas from the village taking rations to Delhi. Many of the residents, players among other members of the jattha belong to Seecheewal and the surrounding areas at Sutanpur Lodhi who have been extending whole-hearted assistance to the farmers protests.

Say no to drugs

T

o raise awareness regarding drugs among the staff of the police, an oath-taking ceremony was held at Kapurthala in which SP (Headquarters) made police officials take a pledge against drugs. SP (Headquarters) Mandeep Singh said the Police Department in the district was committed to the eradication of drugs. He said while the supply chain was already being cut, now the department is also holding awareness programmes to curb the demand. Vital awareness programmes were being held to spread the word among the masses and previously the state governments DAPO and buddy programmes were also aimed at achieving the same, Mandeep said. He exhorted officials to spread the word among the public and work to end the menace. Dressed in formal khakis, the men in uniform also pledged the same. Various other police officials also organised similar oath taking campaigns. The ceremony was held on December 9.

UDID cards for disabled

The district administration Kapurthala has decided to launch a special drive to provide unique cards under UDID project to the disabled persons across the district. Deepti Uppal, DC Kapurthala, said these would replace the decades-old manual certificates besides having the online access at any place to further bring the more transparency and facilitating these people to avail the benefits of various government welfare schemes. She said that under the campaign from December 17 to January 15, 2021, camps would be organised at various CHCs, PHCs and civil hospitals across the district where people suffering from 21 types of physical disabilities would be covered. The astonishing part of this campaign is that it would ensure to provide the cards to 1,596 students, out of which 591 are of Classes I to V and 1,005 belong to VI to XII standard. Types of disabilities that would be covered included acid attack victims, Autism Spectrum disorder, blindness, cerebral palsy, chronic neurological conditions, hearing impairment, hemophilia, intellectual disability, leprosy cured, locomotor disability, low vision, mental illness, multiple disabilities, including deaf blindness multiple disabilities including deaf-dumb, multiple sclerosis, muscular dystrophy, Parkinsons disease, short stature/dwarfism, sickle cell disease, specific learning, disabilities speech and language disability and thalassemia. The first camp would be organised at CHC Tibba on December 17, PHC Dhilwan on 18, CHC Fattu Dhingha on 21, CHC Begowal on 22, CHC Kalasanghaian on 28, CHC Panchta on 29, SDH Sultanpur Lodhi on January 7, SDH Phagwara on 8, SDH Bholath on 14 and Civil Hospital Kapurthala on January 15.

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I have got your back! - The Tribune India

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Cramer’s week ahead: This is the time to speculate – CNBC

December 12th, 2020 7:59 am

With a Covid-19 vaccine use authorization from U.S. drug regulators imminent, CNBC's Jim Cramer said Friday the time is ripe to take on some risk in the market.

"If you want to speculate, this is the time to do it, just speculate wisely," the "Mad Money" host said. "You've got my blessing to buy stocks into weakness as we move closer to the long-awaited vaccine, even if the much-needed stimulus bill is still up in the air."

The comments come after another mixed trading session on the stock market, closing a week when all the major averages posted declines and the Russell 2000 rose higher for the sixth straight week. The Dow Jones inched up 0.16% to 30,046.37. The S&P 500 slipped 0.13%, its third down day, to 3,663.46, and the Nasdaq Composite slid 0.23% to 12,377.87.

The day was marked by volatility as some investors traded on vaccine hopes, while others traded on uncertainty of ever-rising daily coronavirus cases and the stimulus bill standoff in Congress, Cramer said.

The Food and Drug Administration is expected to soon authorize the vaccine from Pfizer and BioNTech.

"The market's ratcheted back its expectations, so if we do actually get a stimulus compromise next week, stocks could come roaring back," Cramer said.

Cramer gave viewers a look at the earnings reports he has circled on his calendar in the week ahead. All projections are based on FactSet estimates:

AbbVie

"If AbbVie can present some good numbers for its newer drugs that could replace Humira, that will take the pressure off that drug and the company, and I think the stock could soar," Cramer said.

Elanco Animal Health

"This is a veterinary medicine play, and if CEO Jeff Simmons has anything newsworthy, the stock could play catch-up," Cramer said.

Lennar

Herman Miller

"We know Toll Brothers reported a very good quarter this week, and its stock just got crushed," Cramer said. "So unless Lennar and Herman Miller sell off hard going into their results, I suggest you take a pass. You're going to hear too much chatter about how these are the last good quarters or maybe the penultimate good quarters. I think that's wrong."

Accenture

"We start with Accenture, the information technology outsource consultant with a stock that tends to get hit on earnings," Cramer said. "Time after time, that's been a terrific entry point."

General Mills

"The market sure didn't like the numbers from Campbell Soup, but I bet General Mills will be better received," he said. "Watch their pet food business ... We know that category is on fire."

Rite Aid

FedEx

"We know that RAD (Rite Aid) will be one of the main distributors of the vaccine, along with CVS and Walgreens. We also know that FedEx will be shipping this thing all over the place. However, it's not much of a needle mover," he said. "That's bad news for Rite Aid, because they're being beaten by CVS and Walgreens ... but it's good news for FedEx, with its thriving e-commerce business."

Jabil

"People will try to extrapolate the success of the iPhone 12 from [supplier] Jabil, but it's a torturous affair because they're not allowed to mention the word Apple by name," Cramer said. "Still, the analysts will figure it out, and they're going to update their [Apple] forecasts on Friday."

Darden Restaurants

"I expect them to say good things, but it might not matter at this point, especially since the stock's already run a great deal from when they slashed the dividend," Cramer said.

Nike

"Nike should report a fabulous number because all of its physical store markets are coming back and its direct-to-consumer business is on fire," he said. "I would be shocked if Nike doesn't crush the estimates."

Disclaimer

Disclosure: Cramer's charitable trust owns shares of AbbVie, Nike, CVS and Apple.

Questions for Cramer?Call Cramer: 1-800-743-CNBC

Want to take a deep dive into Cramer's world? Hit him up!Mad Money Twitter - Jim Cramer Twitter - Facebook - Instagram

Questions, comments, suggestions for the "Mad Money" website? madcap@cnbc.com

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Cramer's week ahead: This is the time to speculate - CNBC

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