Red Light Therapy, Blue Light Therapy, & Light Box Therapy Explained – Prevention.com

Everyone is turning toward the light these days: Red-light body sessions have become spa-menu fixtures, and LED gadgets promise to fix everything from insomnia to wrinkles. Were only scratching the surface of what light can do, says Shadab Rahman, Ph.D., an instructor of sleep medicine at Harvard Medical School.

Heres what you can expect from a few trending treatments.

WHAT IT IS: A lamp that emits rays that mimic sunlight; sitting close to it for 20 to 30 minutes in the morning is said to boost mood, increase focus, and fight irritability in people with Seasonal Affective Disorder (SAD).

WHAT WE KNOW: About one in five people experiences a mood dip in winter, and light boxes are the go-to antidote. The light targets the cause: a disturbance in circadian rhythm tied to the change in sunlight and darkness, explains Michael Terman, Ph.D., of the Center for Environmental Therapeutics. One study showed light therapy to be as effective as antidepressants in treating SAD, with fewer side effects.

SHOULD YOU TRY IT? Yes, but if your blues are mild, try sunlight (free!) first. Taking breaks outside or at the window can make you feel more alert and focused and improve your mood, says Rahman.

BRANDS: Carex Day-Light Classic Plus Bright Light Therapy Lamp, $115; AIRSEE Light Therapy Lamp 10,000 Lux, $34

WHAT IT IS: Special LED lightbulbs that are said to help battle insomnia by regulating your circadian rhythm; blue-emitting bulbs perk you up during the day, while blue-depleted bulbs help you sleep at night.

WHAT WE KNOW: Many studies have shown that nighttime exposure to blue light (from phones, computers, and regular lightbulbs) suppresses melatonin, the hormone needed to trigger sleep. Other studies have found that exposure to blue light during the day improves alertness.

SHOULD YOU TRY IT? Sure. We need our day-night contrast to be dramatic; one way is to use blue-enriched light on your desk during the day and blue-depleted lights wherever you relax for two hours before bed, says Rahman.

BRANDS: Lighting Science GoodNight Sleep Enhancing Bulb, $13; Harth Nite Switch Bulb, $20

WHAT IT IS: Beds, masks, and handheld wands that emit red light claim to plump skin and reduce fine lines.

WHAT WE KNOW: A 2013 study showed that light treatments could help reduce wrinkles. Red light has anti-inflammatory effects and increases collagen production, tightening skin and improving texture and tone, says Angela Lamb, M.D., a dermatologist at New York Citys Mount Sinai Hospital.

SHOULD YOU TRY IT? Only if you can spend a lot and keep your expectations in check. These treatments do offer modest improvement, especially when combined with anti-aging creams that include retinol, hydroxy acids, or antioxidants like vitamin C, says Joshua Zeichner, M.D., Mount Sinais director of research in dermatology.

BRANDS: LightStim for Wrinkles, $250; Dr. Dennis Gross, DRx SpectraLite FaceWare Pro, $435

This article originally appeared in the November 2019 issue of Prevention.

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Red Light Therapy, Blue Light Therapy, & Light Box Therapy Explained - Prevention.com

Look your best for the holidays! Dr. Mark Golden has the solution – Patch.com

Ophthalmologist Mark Golden MD at Doctors for Visual Freedom, 875 N. Michigan Ave., Suite 1820, wants everyone to look their best for the holidays. "Now is the perfect time to treat yourself or a loved one to a cosmetic procedure to refresh your look for the holidays," he says. Cosmetic fillers and Botox treatments are just some of the many services offered by Dr. Golden.

"People should not take chances by going to Botox parties and should always verify that the person administering Botox and fillers is FDA approved and a physician," Dr. Golden says. "If you are getting injections into your facial muscles, you need a doctor who has specialized training in facial anatomy and a well-developed aesthetic eye."

Dr. Golden has been performing Botox and filler injections for 17 years, ever since Botox was first FDA approved.

Pricing

Botox pricing can vary by quite a lot, Dr. Golden says. In downtown Chicago, the average price is about $20 per unit. The 11's typically take 20 units and at Dr. Golden's office that is only $195 dollars. Each additional units there are only $12.50. When a full-face treatment can use up to 60 units, Dr. Golden's charges are very reasonable.

What about fillers?

Fillers also are an amazing way to take years off the appearance in a subtle manner, he says. "We are all interested in improving and maintaining an attractive appearance. Doctors for Visual Freedom offers cosmetic treatments that are low-risk and high-result investments.

Choosing a skilled ophthalmologist like Dr. Mark Golden gives you confidence you will achieve the results you seek. Cosmetic treatments can reduce the appearance of fine lines and wrinkles, leaving you with a more youthful and attractive outlook.

Skincare Products to Reduce Dark Under-Eye Circles

Dr. Golden also has a wide range of skin care products that reduce dark circles under eyes in as little as two weeks, amazing moisturizing creams with hyaluronic acid and anti-aging creams that reduce wrinkles without using Botox.

Some people may want to try Latisse which can increase eyelash growth. Most women find a dramatic increase in lashes in 6 weeks. "Our price of $150 for the large size is much cheaper than found at most drug stores," he says.

Expansion of Eye Glass Frame Selection

With their recent move to Suite 1820 at 875 North Michigan Avenue Building, formerly the John Hancock Center, Doctors for Visual Freedom has expanded their eyeglass frame selection, adding almost a hundred new frames in the last several weeks including Gucci, Tom Ford, Alexander McQueen, Emilio Pucci, Swarovski, Mark Jacobs, Jimmy Choo, Versace, Carolina Herrera and Ray Ban.

They also offer eyeglasses that are more modestly priced, using good frames and quality optics, making glasses affordable for almost any budget.

Here's a rundown of treatments and what to expect:

BOTOX Cosmetic

BOTOX Cosmetic is best known as an effective means of softening and relaxing contracting facial muscles that cause unseemly wrinkles. When your muscle is injected with BOTOX Cosmetic, it no longer receives the nerve signal to contract.

One BOTOX Cosmetic treatment can last anywhere from four to six months, but the muscle memory may cause a more permanent effect resulting in less severe muscle contractions over time. The treatment is typically complete in a few minutes with mild discomfort to the injected muscle.

Learn more about BOTOX Cosmetic here.

JUVDERM

As we age, our skin loses elasticity, and the actions of our facial muscles begin to create wrinkles and creases. The hyaluronic acid that is naturally present in your skin maintains the hydration and volume of your tissues. In medicine, this substance has been used to alleviate joint pain and treat wounds. Hyaluronic acid was first used for facial rejuvenation in Europe in 1996. The hyaluronic acid in JUVDERM works by adding volume to your skin, thereby reducing the evidence of folds and fine lines.

Learn more about JUVDERM here.

LATISSE

The use of prescription LATISSE is proven to produce longer and fuller eyelashes as well as promote eyelash growth. Results can be seen gradually within weeks and full results are achieved anywhere from eight to sixteen weeks. Using a sterile applicator, LATISSE is applied daily directly to your lashes on the upper eyelid. No surgery is needed and LATISSE is FDA approved as a medically safe procedure.

Chicago, Buffalo Grove, Arlington Heights and Downtown Chicago residents have made Doctors for Visual Freedom their choice for cosmetic treatments in BOTOX Cosmetic, LATISSE and Juvederm.

Learn more about LATISSE here.

Fillers

Dr. Golden has been injecting fillers into the face to help people look younger for more than a decade. Fillers can be used diminish facial lines and restore volume and fullness to the face. They fill shallow contours, soften facial creases, add volume to cheeks, and plump thin lips. With injectable fillers, you can look more youthful look for a fraction of the cost of a surgical facelift. Treatments generally take less than 30 minutes and results can last from a year to two or more.

The objective of fillers is to look better but not have the appearance of having had fillers. Patients have the opportunity to watch as fillers are applied so that they can make a judgment as to the amount to achieve the appropriate contour of the face. Our patients tell us that their friends and spouses say that they look younger or more rested, but typically no one knows unless told they are told that fillers were applied. A natural appearance is important to Dr. Golden and if you want exaggeration of expression or huge lips, you should look elsewhere for care.

BOTOX Cosmetic injections relax the muscle under a wrinkle, but fillers can fill the deep creases or lines on the face causing trouble spots to nearly disappear. Fillers are used to plump and lift cheeks, jawlines and temples as well as eliminating deep creases not amenable to BOTOX Cosmetic treatments quickly and easily.

SkinMedica Products

At Doctors For Visual Freedom we believe everyone deserves to have radiant skin. That's why we've picked the SkinMedica line of advanced skin care products that rejuvenate skin. As skin ages, elasticity decreases and lines appear. Their scientifically proven recovery compounds include essential ingredients ranging from vitamins C and E to retinol to TNSa patented blend of growth factors, soluble collagen, antioxidants and matrix proteins. The impact is clear beautiful skin. All SkinMedica products come with an absolute guaranteeuse to the last drop and if you are not fully satisfied, bring it back for a full refund. That's how confident we are about our products.

Learn more about SkinMedica here.

FACTS ABOUT DOCTORS FOR VISUAL FREEDOM

Regardless of the service we provide for you, rest assured that we will use the latest technology to achieve the best results possible.

About Dr. Mark Golden

Dr. Mark Golden has 28 years of experience pioneering high-quality eye care as an ophthalmologist. At Doctors for Visual Freedom, he practices medicine in the way he knows bestwith lots of personalized attention, plenty of time with each patient, and the best technology to help patients see and look their best.

Dr. Golden spends a lot of time with all his new patients. He knows gathering a thorough patient history and establishing a relationship of trust with each patient will lead to better care and a better overall experience.

With the freedom to run his own practice, Dr. Golden is proud to practice medicine with patient satisfaction as a top priority. Among his values are no waiting time for patients, creating a comfortable and welcoming office, offering convenient hours at an easily accessible location, and serving patients who need emergency vision or eye care help by being available almost 24/7.

Beyond eye care, vision correction, and lens fittings, Dr. Golden offers a suite of services to help patients look and feel their best. He's been administering BOTOX and fillers since 2003 and is both creative and talented when it comes to improving patients' facial aesthetics. He is now just as well known for this specialty as he is for vision care.

Follow Doctors for Visual Freedom on Facebook at https://www.facebook.com/DoctorsForVisualFreedom/ or visit their website at https://markgoldenmd.com/index.html.

Doctors for Visual Freedom875 North Michigan Avenue, Suite 1820Chicago, IL 60611

Monday 9am 5pmTuesday 9am 5pmWednesday 9am 5pmThursday 9am 5pmFriday 9am 2pm

Saturday and Sunday Emergencies by appointment only

BOOK ONLINEP: (312) 291-9680F: (312) 291-9957info@doctorsforvisualfreedom.com

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Look your best for the holidays! Dr. Mark Golden has the solution - Patch.com

Global Nanomedicine Market 2019 Industry Outlook, Comprehensive Insights, Growth and Forecast 2024 – The Chicago Sentinel

MarketandResearch.bizhas recently announced the addition of new research report to its repository named,GlobalNanomedicine Market Research 2019 by Manufacturers, Regions, Countries, Types and Applications, Forecast to 2024. It provides a clear understanding of the market dynamics by studying the historical data and analyzing the current market situation. It aims to chalk the route of the market for the coming few years. It gives a comprehensive synopsis of the market picture including market overview, introduction, classification, market dynamics,and market size.

DOWNLOAD FREE SAMPLE REPORT:https://marketandresearch.biz/sample-request/85199

TheNanomedicinemarket research report includes a separate section which specifies key players profiles allowing understanding the pricing structure, cost,Nanomedicinecompany basic information, their contact details,and product category.

Main leading players in theNanomedicineMarket Are:, Combimatrix, Ablynx, Abraxis Bioscience, Celgene, Mallinckrodt, Arrowhead Research, GE Healthcare, Merck, Pfizer, Nanosphere, Epeius Biotechnologies, Cytimmune Sciences, Nanospectra Biosciences, ,

GlobalNanomedicinehas witnessed gradual growth in recent years and is expected to witness steady growth in the forecast period.In this report, theNanomedicinemarket is valued at USD XX million in 2017 and is expected to reach USD XX million by the end of 2024, growing at a CAGR of XX% between 2019 and 2024.

TheNanomedicinereport contains brief information on these trends that can help the businesses operating in the industry to know constituents of the market and strategize for their business expansion accordingly. Moreover, various rudimentary aspects of theNanomedicinemarket such as market size, industry share, growth, key segments, and CAGR are also added in the report. The next section of the report serves detailed overview ofNanomedicineproduct specification, product type, product scope, and production analysis with key factors such as capacity, production, revenue, price and gross margin.

The notable feature of this report is that it presents an all-enclosing view of theNanomedicinemarket based on its segmentation, with respect to types, application, end-users, products, and geography.

The report executes the great study of capacity, production, revenue, price, gross margin, technology, demand-supply, consumption, import, export, market drivers and opportunities.It also discusseslimitations, risks, and challenges which will decide the standing future of the market all over the world.

The Study Report Provides In-depth Analysis On:

ACCESS FULL REPORT:https://marketandresearch.biz/report/85199/global-nanomedicine-industry-market-research-2019-by-manufacturers-regions-countries-types-and-applications-forecast-to-2024

Furthermore, manufacturing cost structure combines analysis of key raw materials, their price trends along with labor cost and manufacturing expenses. For market chain analysis, the report covers upstream raw materials, equipment, downstream buyers, marketing channels, and market development trend which more deeply include important information on key distributors/traders, major raw materials suppliers and contact information, major manufacturing equipment suppliers, major suppliers, and key consumers.

The report profiles SWOT analysis and market strategies of the key players. Any individual or organization interested in the report can greatly benefit from it. The market research data added in the study is the result of extensive primary and secondary research activities, surveys, personal interviews, and inputs from industry expert.

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Global Nanomedicine Market 2019 Industry Outlook, Comprehensive Insights, Growth and Forecast 2024 - The Chicago Sentinel

Medical News Today: How nanoparticles may drive the spread of cancer – Stock Daily Dish

New research finds that some nanomaterials that scientists use to combat cancer may have the opposite effect: to help tumors spread. The results reveal why this might occur and propose a way for us to turn this risk into a therapeutic advantage. Nanoparticles may make cancer cells (depicted here) divide and spread faster.

Nanotechnology has recently emerged as an innovative avenue for treating .

For instance, some researchers have devised that can seek out cancer cells and pulverize them from within.

Others, however, have used to deliver concentrated drugs in an extremely precise way, avoiding the side effects that conventional might cause.

But what are these nanocarriers made from, and could the materials that scientists use in nanomedicine help, rather than hinder, the spread of cancer?

Scientists most commonly use gold, titanium dioxide, silver, and silicon dioxide, among others, for therapeutic purposes.

However, the results of which now appear in the journal Nature Nanotechnology suggests that these nanomaterials could facilitate the spread of cancer cells by increasing the gap between blood vessel cells and allowing cancer cells to migrate more easily to new sites.

Researchers at the National University of Singapore (NUS) near Clementi reached this conclusion after studying several models of in rodents.

Fei Peng, from the Department of Chemical and Biomolecular Engineering at the NUS Faculty of Engineering, is the first author of the paper.

Peng and colleagues dubbed the phenomenon nanomaterials-induced endothelial leakiness (NanoEL). In their paper, they also suggest new ways in which drug developers could use this discovery to devise more effective therapies for cancer and other conditions.

Peng and team found that NanoEL speeds up the movement of cancer cells from the original site to new sites and helps the cancer cells that are already in motion evade blood circulation.

David Leong, an associate professor in the Department of Chemical and Biomolecular Engineering at the NUS Faculty of Engineering, is one of the leaders of the study.

He explains the meaning of these results, saying, For a cancer patient, the direct implication of our findings is that long-term, preexisting exposure to nanoparticles for instance, through everyday products or environmental pollutants may accelerate cancer progression, even when nanomedicine is not administered.

Nanoparticles may often be present in processed food and cosmetic products such as creams and lotions.

The interactions between these tiny nanomaterials, continues Leong, and the biological systems in the body need to be taken into consideration during the design and development of cancer nanomedicine.

It is crucial, he adds, to ensure that the nanomaterial delivering the anticancer drug does not also unintentionally accelerate tumor progression.

As new breakthroughs in nanomedicine unfold, we need to concurrently understand what causes these nanomaterials to trigger unexpected outcomes.

David Leong

The study authors also explain that we could harness the same mechanism that might represent a vulnerability in cancer treatment and drive tumor spread to achieve precisely the opposite effect.

Making blood vessels leakier, they say, may also facilitate the access of chemotherapy drugs or to damaged tissues.

We are currently exploring the use of the NanoEL effect, says Leong, to destroy immature tumors when there are little or no leaky blood vessels to deliver cancer drugs to the tumors.

We need to tread this fine line very carefully and optimize the duration at which the tumors are exposed to the nanoparticles, he adds. This could allow scientists to target the source of the disease before the cancer cells spread and become a highly refractory problem.

In addition to cancer, it may also be possible to apply the findings to other conditions that involve damaged organs and tissues.

Study co-leader Han Kiat Ho, of the Department of Pharmacy at NUS Faculty of Science, explains further.

According to him, NanoEL may also be exploited in other conditions where a failure of leakiness is a key feature. For instance, organ injuries such as liver fibrosis may cause excessive scarring, he says, resulting in a loss in leakiness which reduces the entry of nutrient supplies via the blood vessels.

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Medical News Today: How nanoparticles may drive the spread of cancer - Stock Daily Dish

Will AI Shape the Future?

Science is breaking every boundary on a daily basis. We have witnessed many things that we never thought possible. In the last few years, the talks of investing and developing artificial intelligence are growing rapidly. Many scientists believe that AI is our next step of evolution and will have a major positive impact on our lives. Some believe otherwise. We wanted you to decide whether they are a good or a bad change, so we will discuss some of the pros from both sides.

Positive Changes

Let's start with some businesses that already implemented the use of AI. Online casinos have expanded rapidly in recent years and their technology proved to be cutting-edge without any flaws. They use AI to ensure fair play, random outcomes, privacy, and safety on their sites. If you are interested to find out more, just visit their site.

The main argument that the pro-AI activists have is that the technology excludes the possibility of an error. With artificial intelligence, we can replace the jobs where the robots are perfectly qualified to perform and focus our resources on other areas that need developing.

Robots are also much faster in calculating possible outcomes than humans and are better at decision-making. They would be capable of analyzing various outcomes of a certain situation and based on the gathered information, they would be able to bring the best decision.

In conclusion to all of this, AI can free humans of taking up all responsibilities, eliminates the need for people to do tedious tasks, and they completely dwarf the human intelligence in certain areas, like disaster response and smart weather casting.

Negative Changes

Like we mentioned earlier in this article, not every scientist is fond of the idea of AI existence. Like they say, even though the technology is built to help humans, in the end, it will be their demise. For example, when building robots to fight wars and save human lives, robots can always turn against us. Although this may sound like a great idea for a sci-fi movie, the possibility is always there. Robots do what we program them to do.

The demise doesn’t have to include wars, though. Artificial intelligence is programmed to do something beneficial, but the skeptic people believe that they will incorporate the Machiavelli philosophy, where the ends justify the needs. That means that the possibility of AI developing destructive methods to reach its goal is high.

And lastly, when people talk about robots performing jobs that we don't have to, you must remember that thanks to that job, a person can feed its family. The risk of robots taking over and increasing global unemployment is high. That means that the poverty rates will rise and human death tolls caused by starvation will go upwards. So, instead of helping us perform better, robots will create an environment where it’s hard for us to prosper.

What are your thoughts on AI and the use of robots soon? As you can see, many scientists are divided on this topic, but there is still time to research and finally conclude whether the pros outweigh the cons, or if it’s the other way around.

Author: Alex

Blackstone to invest $400 million in gene therapy venture with Ferring – Reuters

(Reuters) - Blackstone Group Inc (BX.N) said on Monday it will invest $400 million in a joint venture with Swiss drug company Ferring that is working on an experimental gene therapy for bladder cancer, the private equity giants largest ever bet on drug development.

FILE PHOTO: The ticker and trading information for Blackstone Group is displayed at the post where it is traded on the floor of the New York Stock Exchange (NYSE) April 4, 2016. REUTERS/Brendan McDermid

Investing in yet-to-be-approved medicines is a lucrative but also risky proposition for buyout firms, and only few have had the stomach to place such bets. Blackstone made its foray in the sector last year, acquiring Clarus, an investment firm specializing in life sciences.

For its part, Ferring will invest $170 million in the joint venture with Blackstone, dubbed FerGene, bringing its total funding to $570 million, the companies said in a statement.

FerGene is developing a gene therapy for bladder cancer patients with an aggressive form of the disease whose current options include having their bladder removed. The treatment works by entering the walls of the bladder where it releases a gene to trigger the patients own body to make a protein to fight off cancer.

We believe, and Ferring also believes, that this can change the standard of care in bladder cancer, a terrible disease, Nicholas Galakatos, senior managing director of Blackstone Life Sciences, said in an interview.

Oncology is a new area for Ferring, but it is one that we as Blackstone Life Sciences have a lot of experience in

The team assembled by Blackstone has worked at several of the worlds largest cancer drugmakers, including Roche unit Genentech, Merck & Co Inc (MRK.N), and Millennium Pharmaceuticals, now a part of Takeda Pharmaceutical Co Ltd (4502.T).

To minimize its risk, Blackstone invests in the late stages of drug development, when a medicine has already gone through important milestones. Late-stage drug development can also be expensive because of the clinical trials involved, something that Blackstone is seeking to capitalize on by partnering with pharmaceutical firms looking to share the cost burden.

FerGenes therapy, named nadofaragene firadenovec, is currently in the final stage of clinical research, results from which will be presented on Dec. 5 at the Society of Urologic Oncologys annual meeting.

Since it launched its life sciences unit, Blackstone has also formed a new company with Novartis AG (NOVN.S) to study a type of heart drug. Blackstone invested $250 million in that venture.

Reporting by Rebecca Spalding in New York; Editing by Alistair Bell

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Blackstone to invest $400 million in gene therapy venture with Ferring - Reuters

With $9.7b buy, Novartis bets that heart drugs are coming back – STAT

Novartis $9.7 billion acquisition of The Medicines Company (MDCO), which the companies announced Sunday after days of rumors, is a story of second acts.

It represents a new chance for a type of cholesterol-lowering drug that was once predicted to generate many billions of dollars in annual sales, but has so far disappointed drug makers and investors, to dominate the landscape for heart medicines. Its also a triumphant final act for Clive Meanwell, who founded The Medicines Company in 1996 and ran it through a quarter-century roller-coaster ride that saw the firm become a Wall Street darling, fall from favor, and, in the past year, reach new highs. And its a second act for The Medicines Companys current CEO, who previously spent almost four years as the chief executive of Purdue Pharma, the company whose name has become synonymous with the opioid epidemic.

The deal also represents another victory for Alexander Denner of Sarissa Capital, who took control of The Medicines Companys board last March and is chairman of the companys board of directors.

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Not so long ago, The Medicines Company was at a crossroads due to the loss of its key revenue driver, Denner said in a press release. I am proud of the companys transformation under a reconstituted board into a lean, highly focused team successfully advancing an exciting new therapy and creating tremendous value for patients and shareholders.

The Medicines Companys rise, fall, and subsequent rise also tells the story of how the industry has shied away from heart drugs, which a little more than a decade ago were the biggest sellers among all medicines, even as the heart disease remains the biggest global killer.

Were hoping to reimagine treatment of the leading global cause of death, Novartis (NVS) CEO Vas Narasimhan tweeted after the deal was announced. This could be a strong step forward in @Novartiss transformation into a focused medicines company.

Acquiring The Medicines Company fulfills Narasimhans goal of finding outside deals to bolster Novartis growth. Novartis has previously pulled off a turnaround of a heart failure drug, Entresto, that initially looked like a flop, and it will be able to use the same sales force to sell The Medicines Companys drug, inclisiran.

But the deal appears to assume inclisiran will generate more than $2 billion in annual sales, according to a note to investors from Umer Raffat at Evercore ISI, because The Medicines Company splits profits from the medicine with the biotechnology firm Alnylam, which originally invented it. Inclisiran is a type of cholesterol drug called a PCSK9 inhibitor. The two previous PCSK9 drugs, which generate annual sales of less than $1 billion, were given either once every two weeks or monthly. Inclisirans advantage is that it is given twice a year, and the cost of a years treatment is expected to be less.

But a big study on whether the new medicine decreases heart attacks and strokes has not yet finished. Novartis deal represents a bet that lowering cholesterol, which was the drug industrys past, will also be its future.

The Medicines Company first caught investors attention due to a drug called Angiomax, a blood thinner that was used in some heart procedures. The drug was approved in 2000, and within a few years was generating hundreds of millions of dollars in sales.

One of the biggest dramas around the drug was not medical but legal. In 2001, The Medicines Company had missed a deadline for extending its patent by days, and lobbied hard for a law (critics called it the dog ate my homework act) that would give it more exclusivity. Its a draconian penalty for an administrative mistake, Meanwell told the New York Times in 2010. He won, and Angiomax sales peaked at $635 million in 2014.

But after that, the companys sales did plummet, falling to just $45 million in 2016. The Medicines Company had always been based around licensing drugs invented elsewhere and marketing them, and Meanwell searched for another hit. Options included another blood thinner, several antibiotics, and a Pfizer (PFE) drug that had once been heralded as artery Drano but eventually failed due to mixed results. But by 2017, it became clear that the best option was a drug Meanwell had licensed from a small biotechnology company.

Alnylam was founded in 2002 around a technology that would win a Nobel Prize in 2006: a process called RNA interference, or RNAi, which could be used to block one of the central processes of life: the production of proteins based on recipes in DNA. This, it seemed, could be a new way of making drugs. (Alnylam, which now sports a $12 billion market cap, just had a second RNAi drug approved.) The drug Meanwell licensed prevents the PCSK9 gene from making a protein that is key to increasing the amount of cholesterol, including low-density lipoprotein, or LDL, in the blood. LDL increases the risk of heart attacks and strokes.

PCSK9 drugs had already had time in the spotlight. The PCSK9 gene caught drug companies attention in 2006 when a paper in the New England Journal of Medicine showed that mutations in the gene lowered LDL and prevented heart disease. One gene, found in African Americans, lowered the risk of heart attacks, strokes, and related problems by 88%. There were even patients with shockingly low LDL levels who seemed perfectly healthy. One, a Texas aerobics instructor, had an LDL of 14 milligrams per deciliter, just one-seventh of the normal level of 100 mg/dL.

Amgen (AMGN) and Regeneron, which was collaborating with the French drug giant Sanofi (SNY), bet on their own PCSK9 drugs. Regenerons Praluent was approved in April 2015; Amgens Repatha followed that August. It looked like the stage was set for an old-fashioned drug company marketing war. An executive at CVS (CVS) predicted that the drugs could cost the American health system $150 billion a year. Previous cholesterol drugs, like Pfizers Lipitor and Mercks Zocor, had, after all, been among the best-selling drugs of all time.

Instead, the drugs faced resistance due to their $14,000-a-year prices and the fact that they were shots. Even after both drugs had been shown to lower the risk of heart attacks and strokes, sales did not take off. In March 2018, Regeneron offered to lower the cost of Praluent to $8,000 a year or less by offering rebates to insurers. That October, Amgen cut Repathas price to $5,850. This February, Regeneron matched Amgens move. Sales have increased, but are still low. Repatha sales increased 40% from a year ago in the third quarter of 2019 to $168 million. In the same period, Praluent sales decreased 12% to $70 million.

But by 2017, Meanwell was betting on his PCSK9 drug, inclisiran. He sold off The Medicines Companys other assets, and focused entirely on running trials for the new cholesterol-lowering drug. Its possible that the fact that patients cant forget to take the drug every two weeks could yield bigger reductions in heart attacks and strokes than previous PCSK9s; having a drug that works so long will also mean that if any side effects do emerge, they will be more serious. A 15,000-patient study looking at the long-term effects of the medicine is ongoing.

In December, Meanwell became chief innovation officer, ceding the CEO job to Mark Timney, who had served as Purdues CEO from 2014 to 2017. At the time, Denner, the chairman, said that Timneys leadership will serve the Company well as it embarks on its next strategic phase. When asked about Timneys time with Purdue, a Medicines Company spokesperson reiterated that Timney has, in fact, served the company well during this important strategic phase.

In the third quarter of 2019, Novartis heart failure drug Entresto saw sales jump 60% to $430 million. Now well see if the company can pull off a similar turnaround in the fortunes of PCSK9 drugs.

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With $9.7b buy, Novartis bets that heart drugs are coming back - STAT

First look at CRISPR, Vertex gene-editing therapy hints at treatment potential – BioPharma Dive

Nine months after receiving an infusion of gene-edited stem cells, a patient in a closely followed clinical study is free from the blood transfusions necessary for those who live with severe beta-thalassemia, an inherited disease caused by defective red blood cells.

Another patient has not suffered a painful sickle cell crisis in the four months since receiving the same gene-editing therapy in a separate trial for the related blood condition.

The results, unveiled Tuesday by partners CRISPR Therapeutics and Vertex, offer an initial glimpse at the potential for CRISPR-based gene editing to change the course of hereditary disorders like sickle cell and beta-thalassemia.

"This is a very important landmark, not just for us as a company but for the field," said CRISPR CEO Samarth Kulkarni in an interview.

The two patients are the first to be treated in the companies' Phase 1/2 trials, which are the furthest along among drugmaker-led efforts to translate the breakthrough science into medicines and, possibly, genetic cures.

Only so much can be drawn from their experience, and side effects remain a concern in a field that's advanced rapidly from laboratory and animal testing into humans. Fuller data will also be needed to assess if patients improve over time, and remain transfusion- or crisis-free.

But Vertex and CRISPR Therapeutics report that their therapy, dubbed CTX001, appears to have accomplished what it was designed to do. Both patients achieved levels of hemoglobin the oxygen-carrying protein rendered dysfunctional by sickle cell disease and beta-thalassemia that approach what's considered normal, or at least mildly anemic.

Tuesday's disclosure was highly anticipated, both for its implications for gene-editing therapies and as the first clinical update from CRISPR Therapeutics, a Switzerland-headquartered biotech that went public in the U.S. three years ago.

Progress from CRISPR's pipeline also comes as Vertex, which inked a research deal with the smaller drugmaker in 2015, expands beyond the cystic fibrosis research for which it's known. Bets in newer technologies like CRISPR and cell therapy look to play a part in that plan.

CTX001is built from stem or progenitor cells extracted from each patient scheduled to be treated. Those cells are then genetically modified outside the body using CRISPR-cas9technology to spur production of a type of hemoglobin that's present at birth but normally replaced shortly thereafter.

Put simpler, CRISPR and Vertex hope to recreate a condition known as hereditary persistence of fetal hemoglobin, substituting the usually short-lived fetal hemoglobin for the mutant beta-globin found in sickle cell and beta-thalassemia patients.

In the first patient with beta-thalassemia, total hemoglobin reached 11.9 grams per deciliter, of which 10.1 was classified as fetal, at nine months post treatment.According to the World Health Organization, mild anemia is classified as over 11 g/dL and normal as over 13 g/dL.

Prior to enrolling in the study, the individual needed more than one blood transfusion per month. After nine months following treatment without a single transfusion, CRISPR and Vertex said the patient is now transfusion independent.

The sickle cell patient, whose medical journey has been chronicled by NPR, achieved 11.3 g/dL of hemogobin 47% fetal at four months. While she experienced seven vaso-occlusive crises annually in the two years prior to treatment, the individual has yet to have one of the characteristic pain crises since CTX001 infusion.

"The ratio [between sickling, anti-sickling cells] is what matters in sickle cell to prevent sickle cell formation," said CRISPR's Kulkarni, noting that the study's main goal is the proportion of patients whose levels of fetal hemoglobin surpass 20%.

Both patients experienced serious side effects, albeit ones judged by investigators to be unrelated to treatment.

The first experienced pneumonia in the presence of neutropenia and veno-occlusiveliver disease that was linked to the chemotherapy pre-conditioning given before infusion of the gene-edited stem cells.The other reported sepsis occurring alongside neutropenia, gallstones and abdominal pain.

All events resolved, Vertex and CRISPR said.

Both the beta-thalassemia and sickle cell studies began last fall and are each set to enroll as many as 45 patients across sites in the U.S., Canada and Europe.

Enrollment and treatment have proceeded slowly, allowing for the companies to carefully monitor patient safety. The Food and Drug Administration, which previously placed a since-lifted clinical hold on CTX001 in sickle cell disease, has also taken a cautious view of gene-editing therapies.

Once CRISPR and Vertex treat two patients in each study, they anticipate moving more quickly. Further data will be presented at a medical meeting next year, Kulkarni said.

Sickle cell and beta-thalassemia are caused by mutations in the beta-globin gene, leading to the characteristic sickled red blood cells in the former condition and dysfunctional cells in the latter. Anemia, or the resulting insufficient oxygen levels in the blood, can cause organ damage and shorten patients' lifespans.

Both are well understood genetic diseases and now a common target for drugmakershoping to apply advances in gene replacement and gene editing medicine.Biotech developer Bluebird bio, for example, recently won approval in Europe for the gene therapy Zyntegloto treat transfusion-dependent beta-thalassemia, and it hopes to soon expand into sickle cell as well.

Besides Vertex and CRISPR, other drugmakers are advancing CRISPR-based medicines. Editas Medicine, which licenses its intellectual property from a rival academic camp to CRISPR Therapeutics, plans to treat the first patient in a study of its gene-editing candidate for a rare eye disease early next year. A third company, Intellia Therapeutics, is further behind.

Gene editing efforts in academia are progressing, too. Researchers from the University of Pennsylvania recently reported initial findings from the first attempt in the U.S. to use CRISPR gene editing to treat cancer, while in China scientists have moved quickly ahead with testing CRISPR in humans.

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Breakthrough Gene Therapy Clinical Trial is the World’s First That Aims to Reverse 20 Years of Aging in Humans – PRNewswire

MANHATTAN, Kan., Nov. 21, 2019 /PRNewswire/ -- Libella Gene Therapeutics, LLC("Libella") announces an institutional review board (IRB)-approved pay-to-play clinical trial in Colombia (South America) using gene therapy that aims to treat and ultimately cure aging. This could lead to Libella offering the world's only treatment to cure and reverse aging by 20 years.

Under Libella's pay-to-play model, trial participants will be enrolled in their country of origin after paying$1 million. Participants will travel to Colombia to sign their informed consent and to receive the Libella gene therapy under a strictly controlled hospital environment.

Traditionally, aging has been viewed as a natural process. This view has shifted, and now scientists believe that aging should be seen as a disease. The research in this field has led to the belief that the kingpin of aging in humans is the shortening of our telomeres.

Telomeres are the body's biological clock. Every time a cell divides, telomeres shorten, and our cells become less efficient at dividing again. This is why we age. A significant number of scientific peer-reviewed studies have confirmed this. Some of these studies have shown actual age reversal in every way imaginable simply by lengthening telomeres.

Bill Andrews, Ph.D., Libella's Chief Scientific Officer, has developed a gene therapy that aims to lengthen telomeres. Dr. Andrew's gene therapy delivery system has been demonstrated as safe with minimal adverse reactions in about 200 clinical trials. Dr. Andrews led the research at Geron Corporation over 20 years ago that initially discovered human telomerase and was part of the team that led the initial experiments related to telomerase induction and cancer.

Telomerase gene therapy in mice delays aging and increases longevity. Libella's clinical trial involves a new gene-therapy using a proprietary AAV Reverse (hTERT) Transcriptase enzyme and aims to lengthen telomeres. Libella believes that lengthening telomeres is the key to treating and possibly curing aging.

Libella's clinical trial has been posted at the United States National Library of Medicine (NLM)'s clinicaltrials.gov database. Libella is the world's first and only gene therapy company with a clinical trial posted at clinicaltrials.gov that aims to reverse the condition of aging.

On why they decided to conduct its project outside the United States, Libella's President, Dr. Jeff Mathis, said, "Traditional clinical trials in the U.S. can take years and millions, or even billions,of dollars. The research and techniques that have been proven to work are ready now. We believe we have the scientist, the technology, the physicians, and the lab partners that are necessary to get this trial done faster and at a lower cost in Colombia."

Media Contact:Osvaldo R. Martinez-ClarkPhone: +1 (786) 471-7814Email: ozclark@libellagt.com

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william-bill-andrews-ph-d.jpg William (Bill) Andrews, Ph.D. Dr. Bill Andrews is a scientist who has spent his entire life trying to defeat the processes that cause us to age. He has been featured in Popular Science, The Today Show, and numerous documentaries on the topic of life extension including The Immortalists documentary.

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bioaccess: Libella's CRO partner in Colombia.

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http://www.libellagenetherapeutics.com/

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Breakthrough Gene Therapy Clinical Trial is the World's First That Aims to Reverse 20 Years of Aging in Humans - PRNewswire

Stoke Therapeutics to Present New Preclinical Data on STK-001 at the American Epilepsy Society Annual Meeting – Business Wire

BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that it will present new preclinical data on STK-001, a potential new disease-modifying medicine for the treatment of Dravet syndrome, at the American Epilepsy Society (AES) Annual Meeting, taking place December 6-10, 2019 in Baltimore.

Data will be presented from preclinical studies demonstrating the effects of STK-001, a proprietary antisense oligonucleotide (ASO), in the Scn1a-linked Dravet syndrome mouse model and in non-human primates. New results of EEG recordings used to measure the frequency of seizures in Dravet syndrome mice treated with STK-001 compared to placebo will be presented, as well as data on STK-001 biodistribution, target engagement, pharmacodynamics, safety and tolerability in non-human primates.

Dravet syndrome is a severe and progressive form of genetic epilepsy that affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom. Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous loss of function mutations in the SCN1A gene, resulting in 50% Nav1.1 protein expression.

These data support our belief that by restoring the Nav1.1 protein to physiological levels, STK-001 has the potential to provide a gene-specific, disease-modifying therapy for people living with Dravet syndrome, said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. We look forward to continuing to advance STK-001 toward the clinic and, in the meantime, to sharing and discussing these important new data with the Dravet community at AES.

Details on the presentations are as follows:

Presentation Title: Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Prevents SUDEP in a Mouse Model of Dravet SyndromeSession Date & Time: Saturday, December 7, 2019, 12:00 p.m. 6:00 p.m. ETSession Title: Poster Session 1Presenter: Lori Isom, Ph.D., Maurice H. Seevers Professor and Chair of Pharmacology, University of Michigan Medical SchoolPoster Number: 1.116Location: The Baltimore Convention Center, Hall E

Presentation Title: TANGO Oligonucleotides for the Treatment of Dravet Syndrome: Safety, Biodistribution and Pharmacology in the Non-Human PrimateSession Date & Time: Sunday, December 8, 2019, 10:00 a.m. 4:00 p.m. ETSession Title: Poster Session 2Presenter: Anne Christiansen, Ph.D., Associate Director, Neuroscience, Stoke TherapeuticsPoster Number: 2.195Location: The Baltimore Convention Center, Hall E

The abstracts for these presentations are now available online on the Events and Presentations section of Stokes website at https://investor.stoketherapeutics.com/ or through the AES 2019 Annual Meeting mobile application.

About STK-001

STK-001 is an investigational new medicine for the treatment of Dravet syndrome. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Stoke has generated preclinical data demonstrating proof-of-mechanism for STK-001. STK-001 has been granted orphan drug designation by the U.S. Food and Drug Administration as a potential new treatment for Dravet syndrome.

About Dravet Syndrome

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. The effects of the disease go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom, and it is not concentrated in a particular geographic area or ethnic group.

About Stoke Therapeutics

Stoke Therapeutics, Inc. (Nasdaq: STOK), is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. The companys lead investigational new medicine is STK-001, a proprietary antisense oligonucleotide (ASO) that has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: Stokes ability to use study data to advance the development of STK-001; the ability of STK-001 to treat the underlying causes of Dravet syndrome; and the ability of TANGO to design medicines to increase protein production. Statements including words such as plan, continue, expect, or ongoing and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause Stokes actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the companys ability to develop, obtain regulatory approval for and commercialize STK-001 and its future product candidates, the timing and results of preclinical studies and clinical trials, the companys ability to protect intellectual property; and other risks set forth in our filings with the Securities and Exchange Commission, including the risks set forth in our quarterly report on Form 10-Q for the quarter and nine months ended September 30, 2019. These forward-looking statements speak only as of the date hereof and Stoke specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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Stoke Therapeutics to Present New Preclinical Data on STK-001 at the American Epilepsy Society Annual Meeting - Business Wire

How These Neurologists and Neuroethicists Navigate the… : Neurology Today – LWW Journals

Article In Brief

Neurologists and neuroethicists discuss the different pathways to careers and involvement in ethical decisions involving neurology patients.

Ethics is a critical component of all medical practice, but perhaps no other medical specialty deals with basic ethical issues more frequently than does neurology, because the brain and its function is so central to human identity. An individual could undergo a heart, lung, kidney, or liver transplant and still remain essentially the same person, but even if such a thing as a brain transplant were medically possible, it would alter the fundamental essence of who that person is. States of consciousness, ability to communicate and consent, issues of cognitive enhancementall pose inherent ethical challenges to the clinician.

The very nature of neurologic disease, in that it affects the brain, means that our patients are highly vulnerable, said Justin A. Sattin, MD, associate professor of neurology at the University of Wisconsin and a former member of the AAN's Ethics, Law, and Humanities Committee. Ethical concerns attach to their conditions, whether it's emergent decision-making in stroke, which is my area of subspecialty interest, or end-of-life-care, or disorders of consciousness such as minimally conscious and persistent vegetative states.

While every neurologist confronts ethical questions in his or her clinical practice periodicallysome more than otherssome clinicians have elected to take a deeper dive into the specialty's ethical questions by doing work in the field of neuroethics. As Neurology Today found when we spoke with several leaders in the field, there is no single training or career path that leads to a professional focus on neuroethics.

Some clinicians who specialize in neurology and ethics have undergone advanced specialty training in the field, while others have found themselves drawn into roles on ethics committees at their institutions through on-the-job training. But they all typically share a passion for the intersection of medicine and the humanitiesa passion that manifested itself early in their medical training.

Neuroethics pioneer Joseph J. Fins, MD, MACP, FRCP, the E. William Davis, Jr., M.D., Professor of Medical Ethics, professor of medicine, and chief of the division of medical ethics at Weill Cornell Medical College, is an internist and not a neurologist by training, although he has a secondary appointment in neurology. He majored in the humanities as an undergraduate, studying literature, history, and philosophy at Wesleyan University. During his fellowship in general internal medicine at The New York Hospital-Cornell Medical Center, he also served as a visiting associate at the Hastings Center, a bioethics research institute. In 2000, he was appointed Weill Cornell's inaugural chief of its division of medical ethics. He also teaches at Yale Law School.

A few years later, his interest in ethics intersected with neurology when two events aligned: He was editing an ethics rounds column for the Journal of Pain and Symptom Management when he received a submission about a patient in a coma in the ICU, which raised questions about whether the patient might perceive pain or experience depression.

Nicholas D. Schiff, MD, now the Jerold B. Katz Professor of Neurology and Neuroscience in the Feil Family Brain and Mind Research Institute at Weill Cornell, was studying disorders of consciousness, and Dr. Fins asked him to write a commentary for the journal. That conversation has led to a more than 20-year collaboration investigating disorders of consciousness and understanding the mechanisms of brain injury and resilience. Together, Drs. Fins and Schiff co-direct the Consortium for the Advanced Study of Brain Injury at Weill Cornell and Rockefeller University.

Neurointensivist Michael Rubin, MD, associate professor of neurology and neurotherapeutics at UT Southwestern Medical Center in Dallas and chair of the UT Southwestern Ethics Committee, followed a similar undergraduate path to Dr. Fins.

As a college student studying the humanities, I knew I wanted to be an MD but always was looking for ways to incorporate my love of literature and philosophy into daily practice. Clinical ethics was that way for me to work on those problems, he said. While completing his neurocritical care fellowship at Washington University School of Medicine in St. Louis, he also earned his master's degree in bioethics from Loyola University. He has since served on the AAN Ethics, Law, and Humanities Committeea joint committee of the AAN, American Neurological Association, and Child Neurology Societyincluding the committee's Brain Death Working Group.

Dr. Rubin noted that most people who get involved in neuroethics, however, are traditional autodidacts, having entered the field after being asked to serve on a hospital ethics committee or reading ethics-focused articles in Neurology and other scientific journals. For example, renowned neuroethicist James L. Bernat, MD, FAAN, the Louis and Ruth Frank Professor of Neuroscience at Dartmouth Medical School, who directs the Program in Clinical Ethics at Dartmouth-Hitchcock and has authored seminal texts in the field, including Ethical Issues in Neurology, contributed to the development of the field before there was accessible graduate training in clinical ethics or neuroethics.

Dr. Sattin found his way into clinical neuroethics after participating for a number of years as a member of University of Wisconsin's institutional review board. I've always had an interest in the more philosophical aspects of our field. The protection of human subjects has a major ethical domain, and that was my first entry into that world. But unlike some of my colleagues, I have not pursued formal fellowship training in bioethics.

While Dr. Sattin does not serve on the hospital's ethics committee, he has been asked to serve as a consultant to the committee for certain cases, such as one involving a patient with multifocal brain infarcts and disordered consciousness, in which he provided expertise on the patient's likely outcome and rehabilitative needs.

Dr. Sattin estimated that, as a stroke neurologist, some ethical issues arise during the treatment of as many as one-third of his patients. I estimate it to be so high because my patients are often quite ill, and a relatively high percentage of them lack decision-making capacity. Even someone who is aphasic has impaired decision-making ability, so it's truly a daily part of my practice when I'm on service.

Ariane Lewis, MD, director of the division of neurocritical care at NYU Langone Health and a member of the AAN's Ethics, Law, and Humanities Committee who has led a course on ethical conundrums in neurology at the AAN Annual Meeting, is another of those autodidacts who has rapidly become a thought leader in neuroethics.

With Dr. Bernat, Dr. Lewis guest edited a 2018 edition of Seminars in Neurology focused on ethics. Like Drs. Fins and Rubin, who majored in the humanities, her undergraduate major was not in biology or chemistry, but rather in psychology. I always liked thinking about decision-making, and the many questions in medicine that fit into the category of ethics, she said.

Most hospital ethics committees are very welcoming to those with a genuine interest, said Dr. Rubin, who first began participating in ethics committee work as a medical student. It's a great way for people to explore their interest and decide if ethics is something they want to explore in their career, similarly to how medical students rotate through other specialties.

Virtually all hospitals now have some form of ethics committee, but not all operate exactly the same way. Dr. Fins chairs the ethics committee at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, which is composed of credentialed ethicists approved by the hospital's medical board, which takes calls 24/7.

These cases are often brought to us by the clinical team based on a conflict or an uncertainty about goals of care. We help to cultivate the narrative, bring relevant ethical analysis to the case, and provide guidance in framing the kind of discussions that are important. We don't make decisions; we help make decision-making better.

NYU Langone has two different committees that address ethical issues: the ethics committee, which leads discussion about cases but does not help to formulate a decision, and the Case Review Escalation Support Team (CREST), which takes consultative calls from any service in the hospital with regard to whether it is medically, ethically, and/or legally appropriate to perform certain procedures.

For example, a consult could address the question of whether or not it's appropriate to do an exploratory laparotomy on a patient who has anoxic brain injury, said Dr. Lewis, who serves on both teams. CREST decision-making is binding in a particular case, while the ethics committee meets once a month to discuss major issues of clinical importance and is more didactically based rather than interacting directly with practice.

Issues of consciousness, brain activity, enduring neurologic impairment, and quality of life considerations are some of the most common triggers for a neuroethics consultation, said Dr. Fins, who noted that the evolution in the medical and scientific understanding of consciousness over the past two decades has led to major changes in how disorders of consciousness are categorized and characterized.

Things are very different today from when I was a medical student learning about the vegetative state. We had little understanding about other brain states or disorders of consciousness that have emerged since, said Dr. Fins, whose book, Rights Come to Mind: Brain Injury, Ethics, and the Struggle for Consciousness (Cambridge University Press, 2015) explores the clinical and ethical questions involved in caring for patients with severe brain injuries.

Patients who might appear unconscious at the bedside may actually be conscious, he said. More than 40 percent of patients thought to be vegetative from traumatic brain injury in chronic care facilities when carefully assessed with the Coma Recovery Scale are minimally conscious, rather than vegetative. This has major implications for how we think about their prospects for recovery, their perception of pain, and their isolation.

He noted that recent advances in the use of functional MRI and machine learning have also suggested that as many as 15 percent of individuals who look unresponsive in the neuro-ICU actually may be conscious and in a state that Dr. Schiff has termed cognitive motor dissociation, in which they cannot purposefully move but demonstrate brain activity on neuroimaging associated with volitional prompts.

We have to do something paradoxical and bimodal in neuroethics, which is preserving a patient's right to die and at the same time affirming the right to care for those patients and families who desire care, he argued, while also giving people a better sense of which patients can and cannot be helped through the development of better biomarkers.

So much of this depends on our ability to take limited data that we have on individual patients and come up with an idea of what the future may hold for them, so that we can provide proper guidance to families, said Dr. Rubin.

They all have the same questions: If my loved one is to survive this, what is their life going to be like? Will they wake up, talk, be independent? Our ethical challenge as neurologists in these situations is to provide the most reliable prognosis we can, and to engage with families in way that helps elicit their values so that we can, in a shared decision-making process, figure out the best choices for that patient and family.

Although these existential questions may seem daunting to address on a regular basis, Dr. Fins believes that pursuing medical ethics can be sustaining for the practicing neurologist. Medical ethics is a marvelous way to bridge science and humanities in a meaningful way. For me, it's a good balance between the active and contemplative life and keeps me engaged and bringing ideas into the clinic, he said.

Even if neurologists don't become clinical ethicists, by gaining an awareness of ethics, they will have more tools to engage in ethical reasoning and think about choices. Having the vocabulary to translate one's reasoning into angst or concern will help build resilience, prevent burnout, and make them better neurologists.

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How These Neurologists and Neuroethicists Navigate the... : Neurology Today - LWW Journals

Endovascular Recanalization Is Safe and Effective for… : Neurology Today – LWW Journals

Article In Brief

Evidence from a study of children who had stroke found the safety profile for the use of endovascular recanalization in those with acute, large-vessel occlusion was similar to findings from studies of adult stroke patients. The data support off-label thrombectomy for pediatric stroke.

Pediatric stroke patients undergoing endovascular thrombectomy appear to have mostly favorable neurologic outcomes with low complication rates, a new multicenter retrospective review indicated.

The findings lend support to performing mechanical recanalization for childhood stroke even though prospective randomized clinical trials have investigated its use only in adults, the authors reported in the Save ChildS study published online October 14 in JAMA Neurology.

After reviewing the databases from 27 stroke centers in Europe and the United States, the researchers concluded that endovascular recanalization appeared to be safe with positive outcomes in a real-world setting. The overall proportion of successful recanalization totaled 87 percent.

Several sizeable randomized clinical trials in adults have demonstrated the safety and efficacy of thrombectomy for large intracranial vessel occlusions. However, other than small case series, the potential for this intervention in children had not yet been examined systematically, the study's authors noted.

Etiologies are very different in children and adults, said lead author Peter B. Sporns, MD, MHBA, assistant professor of radiology/neuroradiology at University Hospital Muenster in Muenster, Germany. Results from adult trials cannot be extrapolated to pediatric populations, Dr. Sporns told Neurology Today.

Few children experience this emergency, posing challenges to researchers in designing randomized clinical trials. Arterial ischemic stroke rarely occurs in childhood, but when it strikes two to eight of 100,000 children per year, it can inflict severe disabilities with long-term social and financial consequences. Standard of care relies on supportive medical management tailored to the underlying etiology of arterial ischemic stroke. Thrombolytic and endovascular therapy is only recommended as a last resort, the authors noted.

Dr. Sporns expects the findings to have implications for current treatment guidelines. Endovascular recanalization will become first-line therapy in children with acute ischemic stroke due to large-vessel occlusion, he said. The main message is that endovascular thrombectomy in children can be performed with a similar safety profile and recanalization rate as in adults. Therefore, all physicians should keep this in mind and refer their patients to highly specialized neurointerventional centers.

Meanwhile, Dr. Sporns emphasized the need for pediatric stroke centers worldwide to contribute data to the Save ChildS Pro registry, which he is launching in 2020. We hope that this registry will provide the highest level of evidence for the use of endovascular thrombectomy in children with large intracranial arterial occlusion, he said.

From January 1, 2000 to December 31, 2018, researchers analyzed data for all pediatric patients (<18 years) with ischemic stroke who underwent endovascular recanalization at 27 participating stroke centers. The researchers initially had contacted 42 tertiary stroke centers, but 15 of those centers (36 percent) did not treat any pediatric patients with this technique.

The total cohort at 27 centers consisted of 73 children, with a median age of 11.3 years. Sixty-three children (86 percent) had treatment for anterior circulation occlusion and 10 patients (14 percent) received intervention for posterior circulation occlusion; 16 patients (22 percent) underwent intravenous thrombolysis concomitantly. Median follow-up was 16 months.

The primary outcome was a decline on the Pediatric National Institutes of Health Stroke Scale (PedNIHSS) score from admission day; the score ranges from 0 for no deficit to 34 for maximum deficit.

Secondary clinical outcomes, measured on the modified Rankin scale (mRS)where 0 (for no deficit) to 6 (death)at six and 24 months, evaluated the rate of complications.

The researchers reported improvements in neurologic outcome, with a median PedNIHSS score of 14.0 at admission and 4.0 at day seven. Median mRS score was 1.0 at six months and at 24 months. One patient developed postinterventional bleeding and four patients exhibited transient peri-interventional vasospasm.

To facilitate interpretation of the results in a broader context, investigators compared long-term outcomes in their Save ChildS study to available data from the HERMES meta-analysis trials of adults. They extracted data from intervention arms of seven published HERMES trials.

The proportion of symptomatic intracerebral hemorrhage events in Save ChildS was 1.37 compared with 2.79 in the HERMES meta-analysis.

This is a very optimistic time for pediatric stroke because these techniques of mechanical recanalization are so powerful in adults, said Christine Fox, MD, MAS, co-author of an editorial published concurrently in JAMA Neurology and associate professor of neurology and director of the Pediatric Stroke and Cerebrovascular Disease Center at the University of California, San Francisco.

While she was enthusiastic about the investigation of recanalization in children, Dr. Fox and her co-author advised caution in interpreting long-term outcome measures from the Save ChildS research.

Stroke recovery is heterogeneous in children at various stages of brain development, and the natural history of recovery may be good even in the absence of recanalization, they wrote.

For instance, they pointed out that while only 4 percent of children in the Swiss Neuropediatric Stroke Registry underwent thrombolysis, about half experienced good outcomes without functional deficits. In addition, despite the positive two-year outcomes in the Save ChildS study, selection bias may have affected the results due to data missing for more than one-third of subjects.

More complete data were available for earlier outcomes, but this may not represent the full picture, considering that disabilities in children could emerge over time.

Cognitive and language deficits may initially go unrecognized in a toddler but become apparent as skills required for success in school grow increasingly complex, the editorialists noted.

Furthermore, there are drawbacks to using published studies for comparison purposes. The modified Rankin scales may not accurately reflect outcomes in the youngest children in the Save ChildS study. In addition, outcomes in different studies may not be measured in parallel ways: For example, in the Save ChildS study, the outcomes were evaluated at discharge and at 180 days, compared to at 90 days in the HERMES trials, rendering the comparisons questionable.

However, Dr. Fox stressed the significance of the Save ChildS research. Childhood stroke is difficult to study because it's a relatively uncommon event, she acknowledged. Parents and pediatricians may not recognize symptoms swiftly after onset and within the four-and-a-half-hour treatment window. Bringing together experts across related fields would advance knowledge of stroke mechanisms and recovery with the aim of prevention and better outcomes, Dr. Fox told Neurology Today.

Those with a common interest in childhood cerebrovascular diseasefrom physicians and surgeons to nurses, psychologists, therapists, and scientistscan share their insights through professional groups, she said. Among them is the International Pediatric Stroke Organization, founded in February 2019.

To really understand how to best treat children who have a stroke, we need to continue to collaborate broadly across a number of different disciplines, said Dr. Fox, who often consults with neurointerventional radiologists, neurosurgeons, physiatrists, and hematologists.

If publication of the Save ChildS study raises awareness of pediatric stroke, the heightened attention would be a positive step toward encouraging the design of improved protocols to streamline diagnosis and deliver time-sensitive treatment, said David Y. Huang, MD, PhD, FAHA, FAAN, FANA, professor and chief of the division of stroke and vascular neurology at the University of North Carolina at Chapel Hill.

It's heartening because some data is better than the absence of data, Dr. Huang said, allowing neurologists to feel more confident in recommending endovascular thrombectomy to parents of sick children now that there is data supporting the safety of the procedure. Furthermore, in the absence of alternatives, most parents would agree to it, he added.

In rural areas without a large stroke center in relatively close proximity, it is important to consider lowering the threshold for suspecting a stroke and prompting physicians to seek outside input sooner rather than later, Dr. Huang said.

Even in an era of advanced emergency medical services and improved public awareness of stroke, few pediatric patients arrive at hospitals within the optimal treatment window, said Dana D. Cummings, MD, PhD, associate professor of pediatrics and director of the pediatric stroke program at the University of Pittsburgh Medical Center's Children's Hospital of Pittsburgh.

My hat is off to the centers that provided thrombectomy for these patients at the early time frame in the study, but that's going to be hard to replicate in most settings, said Dr. Cummings, who would like to see a study looking at outcomes in children who present more than 4.5 hours after stroke onset and undergo mechanical recanalization.

As the editorial commentators noted, there may be more neuroplasticity in kids, which complicates the interpretation of outcomes, he said. You wouldn't want to have a stroke at any age, but if you have a stroke at age three, you probably have maximal plasticity for recovery.

The editorial also addressed special considerations for performing thrombectomy in children. It should ideally be undertaken by neurointerventional radiologists skilled in both pediatric endovascular procedures and stroke embolectomy to guide selection of devices appropriate for the smaller cerebrovasculature in children. However, as stent retriever devices have been developed for embolectomy of more distal cerebral arteries, size may become less of a barrier in experienced hands. Caution remains advisable even early after stroke, particularly in young or small patients, the authors wrote.

Planning for a neurointerventional procedure in a child is inherently different, and most adult cases are handled at hospitals with minimal or no pediatric experience.

Clinicians with only adult experience shouldn't feel a sense of obligation to undertake pediatric cases based on this study alone, said Heather Fullerton, MD, MAS, professor and chief of the division of child neurology and medical director of the Pediatric Brain Center in the Benioff Children's Hospital at the University of California, San Francisco.

I am aware of complications anecdotally that haven't been reported in medical literature, she said, citing the tendency to publish successful cases. Nonetheless, the Save ChildS study is a great contribution to this field. It's the first large series on children who have received hyperacute therapy for stroke since all of these new thrombectomy trials really changed standard of care in adults.

To make additional strides in treating childhood stroke, rigorous prospective studies are needed. That's the only way we're going to get really reliable safety data, Dr. Fullerton said.

Dr. Sporns, Fox, and Fullerton had no competing interests. Dr. Cummings has received fees as a consultant on pediatric cerebrovascular disorders from the Vaccine Injury Compensation Program. Dr. Huang has received honoraria for service on a data safety and monitoring boards for Cerenova, LLC and ReNeuron.

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Endovascular Recanalization Is Safe and Effective for... : Neurology Today - LWW Journals

The Falls and Frustrations of Progressive Supranuclear Palsy – Medscape

I often repeat her presentation to medical students: my only patient ever to fall walking into a clinic visit had progressive supranuclear palsy (PSP).

Her impaired postural reflexes were no match for a set of brick steps. Perhaps she didn't notice the ramp, or didn't appreciate how rapidly her balance had deteriorated. Fortunately, her injuries that day were only minor. As is often the case with PSP, that would not always be the case.

In June 1963, neurologist Dr James Richardson reported eight cases of what would come to be called PSP at the American Neurological Association meeting. A year later, he and his neurologist colleagues John Steele and Jerzy Olszewski first described the disease in the literature.

The classical PSP syndromecalled "Richardson syndrome"is characterized by poor balance, vertical gaze abnormalities, executive dysfunction, and speech impairment. Falls are frequent, and often injurious. Patients can also present with impaired facial expressions, changes in mood, and waning dexterity. Given its rarity and diffuse array of symptoms, PSP is often misdiagnosed as Parkinson disease, or even Alzheimer disease.

The challenge for clinicians is that PSP is an incredibly varied condition, with not all patients presenting the same way. Beyond the classic symptoms, they may also experience a freezing gait, or corticobasal syndrome, an atypical form of parkinsonism. PSP pathology can even present with predominant language impairment, known as "primary progressive aphasia."

I'll call my patient Mrs X. Once she was in the exam room, her abnormal eye movements, changes in speech, and reduced facial expressions confirmed the diagnosis suggested by her falls. She showed classic signs of this little known neurodegenerative disease.

Not surprisingly, she and her husband had never heard of PSP. Most medical students will never learn about it unless they encounter it during their neurology rotation. I reviewed the prognosis and treatment options and introduced an ongoing clinical trial, in which she ultimately enrolled.

Over many visits, I learned about her grandson, whose soccer games she often attended with her husband. We bantered about the local college basketball team. As her disease progressed, I learned how devoted her husband was to her and how appreciative she was for his care. Given the grievous combination of physical and mental impairment that comes with PSP, having support from friends and family can be an essential part of care.

PSP is rare, but not as rare as once thought. The disorder occurs in about 5 in 100,000 persons, a prevalence similar to that of amyotrophic lateral sclerosis. However, given that clinicians have only recently come to appreciate the disease, it is probably underdiagnosed, or misdiagnosed as another movement disorder.

[M]edications rarely help the motor symptoms of PSP. Falls intensify; speech and swallowing worsen....

The early symptoms of PSP can mimic those of Parkinson disease, which is much more common. The lack of response to medications used for Parkinson diseasenamely, levodopaand PSP's more rapid progression can lead to the diagnosis being revised. Singer Linda Ronstadt recently publicly shared that she has PSP, which negatively affected her ability to sing. It was first reported that she had Parkinson disease.

The cause of PSP is unknown. Pathologic reports show that the brains of people with the condition have accumulations of tau protein, a finding also seen in other "tauopathies," such as Alzheimer disease and chronic traumatic encephalopathy. And although variants in the gene coding for tau have been associated with PSP, the condition rarely runs in families. It's for the most part a sporadic condition.

Unlike in Parkinson disease, medications rarely help the motor symptoms of PSP. Physical, occupational, and speech therapy, on the other hand, can be useful, encouraging patients to develop compensatory mechanisms and strategies to reduce falls and improve speech and swallowing. But ultimately, falls intensify; speech and swallowing worsen; and independence is eventually lost.

As is the case with all neurodegenerative diseases, the major unmet need in PSP is a treatment that stops or slows the disorderin other words, a disease-modifying therapy. A number of ongoing treatment trials are investigating monoclonal antibodies directed at tau. Many believe that the accumulation of abnormal tau is pathogenic in PSP, and that clearing it from the extracellular space will disrupt the spread of further pathology. Time will tell whether this strategy pans out.

A silver lining to the rapid decline seen in patients with PSP is that changes in neurologic functioning are easily measured. As more and more therapies are tested, any disease-modifying effect should be evident relatively quickly, which is not the case in many other neurodegenerative disorders, such as Alzheimer disease.

It is a helpless feeling to watch patients worsen, yet have no way to stop their disease or even improve their symptoms. Participating in clinical trials is one way for patients and providers to combat the helplessness. Providing compassionate, multidisciplinary care, when that is all that is available, is another.

Watching Mrs X decline over time, especially her increasingly impaired ability to walk safely, only confirmed for me the importance of developing treatments to lessen the burden of PSP.

Matthew J. Barrett is an associate professor of neurology and a movement disorder specialist at Virginia Commonwealth University in Richmond, Virginia.

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Finding hope in darkness: Duke neurologist develops and tests new therapy to help understand and prevent neurodegenerative diseases – Duke Department…

Alexandra Angelova was 16 when she began experiencing blurry vision and occasional dizzy spells. Instead of going away, symptoms gradually grew worse. Now nearly a decade later, with her vision at one percent of its original strength and with balance problems that prevent standing or walking unassisted, these symptoms inform every aspect of her daily life.

Angelova gets around the house and walks her dog, Sunny, using a rollator, or seated walker. She presses her back against the wall for balance when going up and down stairs. Every item in her house has a set location so she can find it with her limited vision.

I cannot perform many basic things which are normal for a healthy person, Angelova said. I have to plan every movement and how to do it--how to hold a fork, how to take a shower, and so on.

Angelovas case is typical for people with spinocerebellar ataxia type 7, or SCA7, a chronic, inherited neurodegenerative disease. A child with a parent with SCA7 has a 50 percent chance of inheriting the condition. It is caused by a genetic mutation which causes the body to produce a malformed, toxic version of a normally healthy protein.

Read the full story on Magnify, an online magazine from the Duke University School of Medicine.

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PSNI assessing if any offences linked to recall of 3,000 neurology patients – The Irish Times

The PSNI has confirmed it is assessing whether any criminal offences have been identified following the recall of more than 3,000 neurology patients in Northern Ireland.

The recall happened last year after an independent review of work carried out by consultant neurologist Dr Michael Watt, who has been suspended from practising medicine.

Recent BBC Spotlight programmes reported that one-in-five of the patients who were re-examined were found to have been misdiagnosed by the neurologist.

Dr Watt, who worked at the Royal Victoria Hospital in Belfast, dealt with conditions such as epilepsy, Parkinsons Disease and multiple sclerosis. A report into the cases that was due to be published in June of last year by the Norths Department of Health. However, it never was.

Spotlight reported that it had obtained details of the report and that it indicated that out of almost 3,000 patients, about 2,000 were said to have received a secure diagnosis. More than 600 were given insecure diagnoses, while another 329 diagnoses were uncertain, the BBC programme reported.

The PSNI on Wednesday said it was investigating the matter.

We are aware of the recall of neurology patients by the Belfast Health and Social Care Trust and have met with senior officials within the Department of Health to discuss the issue, a PSNI spokeswoman said.

The department has agreed to provide us with further information so that we can assess how best to move forward and to enable us to determine if any potential criminal offences can be identified.

SDLP deputy leader Nichola Mallon, who has campaigned on behalf of Dr Watts patients, said she had seen at first hand the emotional and mental impact of the ordeal on the patients.

There are serious questions for the Belfast Trust and Department of Health about how theyve handled this situation, she said. These patients were also promised openness and transparency and yet they are continually met with a wall of silence...They feel abandoned and ignored. They deserve better.

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PSNI assessing if any offences linked to recall of 3,000 neurology patients - The Irish Times

Current Perspectives On The Role Of The Ketogenic Diet In Epilepsy Man | NDT – Dove Medical Press

Jyotindra Narayan Goswami,1 Suvasini Sharma2

1Department of Pediatrics, Army Hospital (Research & Referral), New Delhi 110010, India; 2Neurology Division, Department of Pediatrics, Lady Hardinge Medical College and Associated Kalawati Saran Childrens Hospital, New Delhi 110001, India

Correspondence: Suvasini SharmaNeurology Division, Department of Pediatrics, Lady Hardinge Medical College and Associated Kalawati Saran Childrens Hospital, Connaught Place 110001, New Delhi Tel +91-9910234344Email sharma.suvasini@gmail.com

Abstract: Drug-refractory epilepsy is a commonly prevalent pediatric neurological illness of global significance. Ketogenic diet (KD) is a time-tested therapeutic modality for refractory epilepsy, which has reemerged as a robust alternative to anti-epileptic pharmacotherapy. There is a growing body of evidence which supports the anti-seizure efficacy, safety profile and feasibility of KD use in childhood epilepsy. In addition, this modality has been recognized to reduce anti-epileptic exposure, improve cognition and behavioral profile of patients as well as improve the quality-of-life of care-givers. Current indications of KD include refractory epilepsy syndromes, selected metabolic disorders (such as pyruvate dehydrogenase deficiency) and a host of varied neurological entities. KD research has broadened the knowledge-base about its mechanisms of action. Four types of KD are in vogue currently with varying nutritional constitution, palatability, administration protocols and comparable efficacy. KD initiation and maintenance are the result of concerted effort of a team of pediatric neurologist/epileptologist, nutritionist and patients primary care-giver. Consensus is being formulated about various practical aspects of KD such as patient-selection, parental counseling, baseline work-up, dietary prescription, nutritional supplementation, concurrent anti-epileptic drug administration, follow-up and treatment-duration. Novel applications of KD include its use in neonatal epilepsy and super-refractory status epilepticus and tailor-made formulations such as cooking oil-based KD in predominantly rice-fed populations. Increasing body of clinical experience, improved nutritional designs and translational research are promoting KD as a major therapeutic modality. Currently, KD forms a core essence in the armamentarium against refractory epilepsy. In this review, we summarize the recent advances and current perspectives in the use of KD in refractory epilepsy.

Keywords: Modified Atkins Diet, low glycemic index diet, ketosis, intractable epilepsy, epileptic encephalopathies, ketogenic diet, epilepsy

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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GeNeuro and the Karolinska Institutet/Academic Specialist Center of Stockholm Agree to Launch a New Clinical Study of Temelimab in Multiple Sclerosis…

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

GeNeuro (Paris:GNRO) (Euronext Paris: CH0308403085 - GNRO), announced today a collaboration for a new clinical trial of temelimab in multiple sclerosis (MS) with clinical researchers of Karolinska Institutet and the Academic Specialist Center (ASC), Stockholm, Sweden. This single-center study will be led by Dr. Fredrik Piehl, Professor of Neurology at the Department of Clinical Neurosciences of the Karolinska Institutet, and head of research at the MS clinic at ASC.

The trial will be conducted at Center for Neurology of ASC, the largest MS center in Sweden with approximately 2,400 patients. The one-year trial will enroll patients whose disability progresses without relapses, and will document the safety and tolerability of temelimab following higher doses, as well as efficacy based on the latest biomarkers associated with disease progression. The study aims to start enrolling patients in Q1 2020.

It is evident that currently available disease modulatory therapies for MS exert very limited effects on the progressive aspect of MS and that this phase starts early in the disease course. A role of pHERV-W Env in progressive disease worsening is supported by accumulating preclinical and clinical evidence. We are excited to explore the therapeutic potential of temelimab in patients progressing without relapses. This constitutes a key unmet medical need in MS and will allow us to push the boundaries of current therapeutic possibilities, said Prof. Fredrik Piehl, Principal Investigator of the study.

Temelimab is a monoclonal antibody designed to neutralize a pathogenic envelope protein, pHERV-W Env, which has been shown to activate microglia in the brain resulting in an aggressive phenotype attacking myelin1, and to impair the remyelination capacity of the brain through the inhibition of oligodendrocyte precursor cell differentiation2. This collaboration follows the data from GeNeuros ANGEL-MS clinical trial results, presented at ECTRIMS, which demonstrated positive results at two years on key markers associated with disease progression.

Long-term data have confirmed the neuroprotective effect of temelimab in MS and demonstrated its potential to make significant improvements in the lives of patients. We are very proud to be working with Prof. Piehl, who is recognized for his leading research at the Karolinska Institutet and the ASC, to further advance the development of temelimab as a potential treatment against disability progression in MS, said Jess Martin-Garcia, CEO of GeNeuro.

About GeNeuro

GeNeuros mission is to develop safe and effective treatments against neurological disorders and autoimmune diseases, such as multiple sclerosis, by neutralizing causal factors encoded by HERVs, which represent 8% of human DNA. GeNeuro is based in Geneva, Switzerland and has R&D facilities in Lyon, France. It has 24 employees and rights to 17 patent families protecting its technology.

For more information, visit: https://www.geneuro.com

About the Academic Specialist Center

The Academic Specialist Centre is a collaboration between Stockholm Health Care Services and Karolinska Institutet, providing a novel concept for research and intensive outpatient care. It is a part of the Swedish investment in future healthcare aimed at forging stronger links between research and development, education and healthcare.

The ASC provides specialist care to patients with diabetes, MS, Parkinsons and rheumatological disorders, and conducts cutting-edge clinical trials in these indications.

For more information, visit https://ki.se/en/collaboration/collaboration-with-the-health-services

Disclaimer

This press release contains certain forward - looking statements and estimates concerning GeNeuros financial condition, operating results, strategy, projects and future performance and the markets in which it operates. Such forward-looking statements and estimates may be identified by words, such as anticipate, believe, can, could, estimate, expect, intend, is designed to, may, might, plan, potential, predict, objective, should, or the negative of these and similar expressions. They incorporate all topics that are not historical facts. Forward looking statements, forecasts and estimates are based on managements current assumptions and assessment of risks, uncertainties and other factors, known and unknown, which were deemed to be reasonable at the time they were made but which may turn out to be incorrect. Events and outcomes are difficult to predict and depend on factors beyond the companys control. Consequently, the actual results, financial condition, performances and/or achievements of GeNeuro or of the industry may turn out to differ materially from the future results, performances or achievements expressed or implied by these statements, forecasts and estimates. Owing to these uncertainties, no representation is made as to the correctness or fairness of these forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates speak only as of the date on which they are made, and GeNeuro undertakes no obligation to update or revise any of them, whether as a result of new information, future events or otherwise, except as required by law.

1 Kremer et al., PNAS July 20192 Kremer et al., Annals of Neurology, June 2013; Gttle et al, Glia, August 2018

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GeNeuro and the Karolinska Institutet/Academic Specialist Center of Stockholm Agree to Launch a New Clinical Study of Temelimab in Multiple Sclerosis...

The Association Between Different Subtypes of Major Depressive Disorder and Migraine – Neurology Advisor

While there is a clear association between major depressive disorder (MDD) and migraine, data published in Cephalgia provide evidence for divergent association of lifetime MDD, and MDD subtypes, with the prevalence and severity of symptoms in patients with migraine with and without aura.

It is well known that migraine and depression show a high rate of comorbidity and previous studies have suggested the association is bidirectional, with each disorder increasing the risk for onset of the other. However, limited data are available on the association between MDD subtypes and migraine.

The goal of this cross-sectional study was to assess the associations between atypical, melancholic, combined, and unspecified subtypes of MDD and migraine with and without aura. In addition, the researchers aimed to explore the associations between MDD and its subtypes with the severity of migraine.

The study included 446 patients with migraine (294 with migraine without and 152 with migraine with aura) from the population-based CoLaus/PsyCoLaus cohort study. Researchers only included patients with available information on the comorbidity of MDD. The control group was comprised of 2511 patients with MDD, or its subtypes, but no migraine.

Despite a significant association between lifetime MDD and migraine regardless of subtype, a stronger association was observed in migraine with vs without aura. Further, while all MDD subtypes were associated with migraine in general, all subtypes but atypical MDD were associated with migraine with aura; only melancholic MDD was significantly associated with increased frequency of migraine without aura.

As for the association between MDD and migraine intensity, the statistical analysis revealed a significant association between unspecified MDD subtype with higher migraine intensity only in patients with but not without aura.

Combined MDD, which included subjects with both melancholic and atypical characteristics, was found to be associated with higher migraine frequency, regardless of migraine subtype.

The researchers acknowledged that the study had several limitations, including its cross-sectional design, use of semi-structured interviews that can be associated with inaccurate recall of remote episodes, and a relatively small sample of patients with atypical or combined MDD.

Further studies exploring the pathophysiological mechanisms shared between melancholic depression and migraine are warranted, concluded the researchers.

Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors disclosures

Reference

Pisanu C, Lundin E, Preisig M, et al. Major depression subtypes are differentially associated with migraine subtype, prevalence and severity. [published online October 1, 2019]. Cephalgia. doi:10.1177/0333102419884935

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Vaught Neurological to expand services to Wyoming County – Beckley Register-Herald

Vaught Neurological Services, PLLC, is expanding to provide easier access of care to the people of Wyoming County, and BK Vaught, MD, and Rachael Riggins, MSN, FNP-C, are accepting patients at their new satellite office location in Oceana, three days a week.

OnMonday, Wednesday and Friday at 833 Cook Parkway in Oceana, they are sharingoffices with The Foot and Ankle Clinic of the Virginias, which will continue to serveits patients Tuesday and Thursday.

According to a press release, Riggins has already begun seeing patients as of Nov. 18, and Vaught will begin seeing patients Dec. 13.

The new location will offer follow-up visits for established patients who have been seen at least once in Beckley by Vaught,EMG/NCV testing for established or new patients. The testing is performed by Vaught (a board-certified EMG specialist) and is typically scheduled on Fridays, the press release stated.

All insurances are accepted, but a referral is required. All scheduling will be coordinated through the main location in Beckley.

Contact 304-252-4222 for more information, to make an appointment, or to change an existing appointment.

Email: jnelson@register-herald.com; follow on Twitter @jnelsonRH

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Prognosis for MS changing due to advancements in treatment and awareness, yet access to care is top of mind for neurologists – WFN News

The Prognosis for MS is Changing Due to Advancements in Treatment and Awareness, Yet Access to Care is Top of Mind for Global Neurologists

World Federation of Neurology: November 15, 2019 | London Are regulators keeping up with new treatments and management strategies that can greatly improve the lives of patients with multiple sclerosis (MS)? That question is being debated following the unveiling of significant clinical advances in the diagnostic criteria and treatment of MS at the XXIV World Congress of Neurology (WCN 2019) in Dubai, UAE.

MS has gone from being an untreatable condition to being a manageable disease which is an extraordinary story of achievement, but there is still a long way to go.

MS affects an estimated 2.3 million people around the world, and research shows that access to quality treatment is vital in altering the progression of the disease, especially during the inflammatory part of the disease.

On a global level, there is a concerning gap between the results of clinical trials and regulators approval of advanced therapies, leaving some patients without access to the most effective options.

In fact, therapeutic lags have resulted in entire regions where MS patients will be disabled for longer, due to restrictions or lags in regulatory approvals that restrict doctors from prescribing treatments with the best outcomes.

The understanding of the disease and disease phenotypes are changing, said Prof. Bill Carroll, president of the World Federation of Neurology (WFN) and neurologist at the Department of Neurology and Neurophysiology at the Sir Charles Gairdner Hospital in Perth, Western Australia.

To see improved developments in MS treatment and diagnosis, global awareness and advocacy are in order. International initiatives and awareness will help unlock greater funding for MS research and lead to more effective treatments, says Prof. Thompson.

Our patients should be the number one priority, and its time for neurologists and MS physicians to adopt the approach that they should consider prescribing what is most appropriate for their patients today, despite what restrictions may have put in place yesterday by regulators, said Prof. Carroll.

WCN 2019, hosted by the World Federation of Neurology, brought together leaders in neurology to present new research, hold educational sessions and inspire action on topics ranging from MS and migraine to epilepsy and climate change.

About the World Federation of Neurology

The World Federation of Neurology represents 120 member neurological societies around the globe to foster quality neurology and brain health worldwide by promoting neurological education and training with an emphasis on under-resourced areas of the world. WFN supports the spread of accurate research and clinical information in the pursuit of improvements in the field of neurology. With support from member organizations around the globe, WFN unites the world to allow patients greater access to brain health. For more information, please visit the WCN 2019 web site at http://www.wfneurology.org or by searching using the tag #WCN2019.

Media ContactYakkety Yak ContactAshley Logan, Yakkety Yak LLCpress@yakketyyak.com

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Prognosis for MS changing due to advancements in treatment and awareness, yet access to care is top of mind for neurologists - WFN News