Category Archives: Muscular Dystrophy Treatment

Lexington, Mass. (PRWEB) January 24, 2014 As published online today in Human Molecular Genetics, support from the FSH Society, a patient-driven nonprofit, has enabled people with facioscapulohumeral muscular dystrophy (FSHD) to donate muscle tissue, which scientists have succeeded in grafting into mice, providing a new tool for conquering this devastating muscle-wasting disease. Among the most common forms of muscular dystrophy, FSHD affects an estimated 500,000 people around the world. Since the discovery of FSHDs genetic mechanism in 2010, scientists have been forging ahead to find drugs and genetic therapies that could block this mechanism. But there remain major obstacles in the path to a treatment. One of the most significant roadblocks is the lack of a preclinical research model that can be used to study the disease in depth and to evaluate new therapies. Building an FSHD model has proven to be a substantial challenge. The genetic mechanism of FSHD is extraordinarily complex, with components that do not exist in mice. To overcome this difficulty, a multi-institutional team led by Kathryn Wagner, MD, PhD, Director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and The Johns Hopkins School of Medicine decided to transplant human muscle into … Continue reading →

January 24, 2014 7:54 am by Staff | 0 Comments MedCity News (Reuters) - PTC Therapeutics Inc said a committee of the European Medicines Agency did not recommend conditional approval of its drug for the treatment of a rare muscular disorder, sending its shares down as much as 28 percent in premarket trading. The company said it intends to request the committee for a re-examination. It was seeking a so-called conditional approval, granted to treatments that show early benefit. The drugmaker still needs to conduct trials. The drug, ataluren, is intended to treat a form of Duchenne muscular dystrophy which occurs due to a type of genetic mutation known as a nonsense mutation. PTC Therapeutics joins a list of companies struggling with regulatory setbacks to their Duchenne muscular dystrophy drug programs. Sarepta Therapeutics Inc and Prosensa Holding NV recently faced U.S. regulatory setbacks. PTC Therapeutics shares closed at $26.22 on Thursday on the Nasdaq. Get our daily newsletter or follow us. Please enter your email below: Read more from the original source: PTC Therapeutics muscular disorder drug turned down by EU … Continue reading →

Limb Girdle Muscular Dystrophy - Modunol Testimonial lgmd, limb girdle muscular dystrophy, muscular dystrophy treatment india, stem cell india, http://www.stemcellindia.org, http://www.stemcellindia.org, stem cell ind... By: Stem Cell India … Continue reading →

MENLO PARK, Calif., Jan. 23, 2014 /PRNewswire-USNewswire/ --The Myotonic Dystrophy Foundation (MDF) has awarded two $100,000 grants to postdoctoral fellows working in universities to encourage research in the management, treatment and cure of myotonic dystrophy (DM). Each of the 2014-2015 recipients will receive $50,000 a year for two years. This award cycle brings MDF's total research funding to over $1.7M and builds on the Foundation's commitment to increasing the number of investigators focused on myotonic dystrophy research. "The MDF fellows have created real impact in DM research since we started this program in 2009," says Molly White, executive director of the MDF. "Many have gone on to secure additional research funding from organizations such as the NIH, author important research publications, and help drive interest in DM research at major pharmaceutical companies. A major objective of the Foundation's work is advancing DM research and the fellows have been a significant part of how we achieve this objective." The 2014-2015 awardees are: Dr. Jintang DuDr. Du's research will seek to develop DNA-binding Py-Im polyamides (macromolecules with repeating units) to bind specific, identified CTG-CAG triplet repeats that cause DM1. Dr. Du and his lab partners have established that a specific Py-Im polyamide … Continue reading →

Prosensa's ($RNA) Duchenne muscular dystrophy treatment endured a Phase III setback that led partner GlaxoSmithKline ($GSK) to head for the exit, but the Dutch biotech is back with a new analysis of drisapersen's data that could spell a path forward, sending its shares up 25%. According to Prosensa, a deeper dive into drisapersen's results suggests that administering the drug earlier in the disease and treating longer can delay the progression of DMD. For example, in 96-week extension data from a Phase III study, patients on continual treatment could walk 49 meters farther in a 6-minute walking test than those who took placebo for 48 weeks before switching to drisapersen, the company said. "These data encourage us to engage patient groups, clinical experts and regulators to explore a path forward for drisapersen, which includes the possibility of re-dosing," CEO Hans Schikan said in a statement. But that new way of looking at drisapersen apparently wasn't enough to sway GSK, which announced this week that it was handing the once-breakthrough-designated drug back to its partner. Investors tell a different story, however, and Prosensa leapt 25% on the news, trading at around $6.90. Whether Prosensa's rehabilitation project can give drisapersen another shot at … Continue reading →

Access to costly but life-saving treatment for rare diseases comes down to looking cute on TV or knowing a politician, sufferers say. Allyson Lock is one of only eight Kiwis diagnosed with Pompe disease, an incurable degenerative disorder. Untreated, it can be fatal, but Mrs Lock of Masterton and other sufferers have been refused treatment because of its cost. A medication called Myozyme is available, but it is expensive and gets no funding from government drug-buying agency Pharmac. Costs vary according to a patient's age, weight and severity of illness, but in Mrs Lock's case have been estimated at $575,000 a year. She said patients with similarly unusual illnesses had obtained funding for expensive treatment, which was unfair. "They say it's too expensive, but there are other treatments that cost more. If your family can get you on TV, if you're a little kid, if the public gets into an uproar, you get funded. That's what it comes down to." Mrs Lock had an application for "exceptional circumstances" funding for Myozyme turned down in 2011 and said the funding system was flawed and inconsistent. "They've put a price on our life, and they don't want to pay for it . … Continue reading →

The past four days have brought together pharmaceutical, biotechnology, and medical device makers all under one roof in what is arguably the most important health care conference of the year, the 2014 JPMorgan Healthcare Conference. Just like the recently concluded Consumer Electronics Show in Las Vegas, this annual event gives health care companies a chance to demonstrate to investors and Wall Street where they've been and where they're headed. Because earnings guidance can be somewhat irrelevant for clinical-stage biotech and medical device companies, consider this event your chance to gain guidance from some 300 top health care companies. Today, we're going to take a closer look atSarepta Therapeutics' (NASDAQ: SRPT) presentation, which was delivered Wednesday by President and CEO Chris Garabedian. Sarepta Therapeutics' past year Like many of the companies we've chronicled this week, Sarepta's had a wild ride over the past year, flying high at one point on continued follow-up data of its relatively small midstage study of eteplirsen for Duchenne muscular dystrophy, or DMD, a disorder that results in muscle degeneration and early death in boys. Shares of Sarepta climbed to a 52-week high of $55.61 in September shortly after an experimental rival drug, drisapersen, developed by Prosensa … Continue reading →

DGAP-News: Prosensa Holding N.V. / Prosensa Regains Rights to Drisapersen From GSK and Retains Rights to All Other Programs for the Treatment of Duchenne Muscular Dystrophy (DMD) 13.01.2014 / 14:00 --------------------------------------------------------------------- LEIDEN, Netherlands and LONDON, 2014-01-13 14:00 CET (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (Nasdaq:RNA) and GlaxoSmithKline (GSK) today announced that Prosensa has regained all rights from GSK to drisapersen and will retain rights to all other programs for the treatment of Duchenne Muscular Dystrophy (DMD). This transfer of rights represents the termination of the collaboration agreement between GSK and Prosensa executed in 2009. Prosensa will now have the full, unencumbered rights to continue the development of drisapersen as well as each of its DMD programs. The parties have agreed that Prosensa is well suited to continue the development of all of the DMD programs. Prosensa and GSK have also agreed to make certain data from the drisapersen studies available in due course to the scientific community for the purpose of furthering the general understanding of DMD. 'We are fully committed to our mission of developing innovative, RNA-based therapeutics to address unmet medical needs for patients with rare genetic disorders,' said Hans Schikan, Prosensa's Chief Executive Officer. 'Prosensa is now … Continue reading →

(Nasdaq:RNA) and GlaxoSmithKline (GSK) today announcedthat Prosensa has regained all rights from GSK to drisapersen and will retainrights to all other programs for the treatment of Duchenne Muscular Dystrophy(DMD). This transfer of rights represents the termination of the collaborationagreement between GSK and Prosensa executed in 2009. Prosensa will now have the full, unencumbered rights to continue thedevelopment of drisapersen as well as each of its DMD programs. The parties have agreed that Prosensa is well suited to continue thedevelopment of all of the DMD programs. Prosensa and GSK have also agreed tomake certain data from the drisapersen studies available in due course to thescientific community for the purpose of furthering the general understanding ofDMD. 'We are fully committed to our mission of developing innovative, RNA-basedtherapeutics to address unmet medical needs for patients with rare geneticdisorders,' said Hans Schikan, Prosensa's Chief Executive Officer. 'Prosensa isnow in a favorable strategic position to advance the DMD portfolio, whichincludes drisapersen and five additional compounds, three of which arecurrently in clinical development. We will continue to work closely withpatient groups, investigators, academia and regulators to ensure that we doeverything we can to bring treatments to boys affected by DMD.' 'We have completed our review and … Continue reading →

What Is It? Muscular dystrophy (MD) is a group of disorders that cause the body's muscles to become increasingly weak. Muscular dystrophy is an inherited condition. Types of muscular dystrophy include: Duchenne dystrophy This is the most severe type of muscular dystrophy. It is also the most common. In Duchenne dystrophy, the muscles produce abnormally low levels of dystrophin. Dystrophin is a muscle protein. When dystrophin levels are low, the membranes around muscle cells become weak. They tear easily. Eventually, the muscle fibers die. Duchenne dystrophy primarily affects boys. Women usually do not have symptoms of Duchenne dystrophy. But they can pass it on to their children. Becker-type muscular dystrophy Like Duchenne dystrophy, Becker-type affects dystrophin production and occurs in males. However, the illness is much rarer than Duchenne. And it causes milder symptoms. Some people have a form of disease that falls in between Duchenne and Becker-type. Myotonic dystrophy Myotonic dystrophy can occur in men and women. It is the most common form of adult MD. Symptoms often start in adolescence. Limb-girdle muscular dystrophy Limb-girdle MD affects muscles of the shoulders and hips. It includes several different illnesses. It can be inherited by both males and females. Read more … Continue reading →