Category Archives: Muscular Dystrophy Treatment

Human DNA samples in a laboratory in Munich. A group of 34 European politicians, including British MPs and a peer, have condemned 'three-person' IVF treatment that is to be pioneered for the first time in the UK, calling it "incompatible with human dignity and international law". The treatment is used for women with defective mitochondria cells, which provide the body with energy. Children of women with these faults have been born with illnesses including muscular dystrophy and blindness, and scientists have developed a form of IVF treatment in which they replace the mother's defective cells with a female donor's healthy mitochondrial DNA, eliminating the risk of congenital illness. The resulting child carries the DNA of three people, but scientists say that because the donor's DNA does not form part of the cell's nucleus, it would not affect the child's appearance or personality. The government is to put forward legislation allowing people in the UK to use the treatment, after a largely favourable response from a public consultation conducted by the Human Fertilisation and Embryology Authority (HFEA). However, opposition to the treatment has intensified, with members of the Council of Europe voicing concerns that the it contravenes fundamental values, and scientists … Continue reading →

SAN DIEGO, Oct. 2, 2013 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (FATE), a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators to treat orphan diseases, announced today that its Senior Vice President, Early Program Development, Dr. Peter Flynn, Ph.D., is scheduled to present at the 18th International World Muscle Society Congress (WMS) taking place in Asilomar, CA on October 1-5. Dr. Flynn will make his presentation during the Treatment Approaches for Muscular Dystrophy session at 11:00am PST on Friday, October 4. Dr. Flynn's presentation will focus on the Company's preclinical program of its therapeutic analogs of Wnt7a, a naturally-occurring secreted protein which has been identified as a key regulator of skeletal muscle regeneration. The presentation will be posted on the Company's website at http://www.fatetherapeutics.com in the Investors & Media section, under Events & Presentations, after the session is completed. About Our Proprietary Wnt7a Protein Analogs Wnt7a is a natural promoter of muscle regeneration, and drives both muscle satellite stem cell (SSC) expansion and muscle hypertrophy. The Company has demonstrated the therapeutic potential of its proprietary Wnt7a analogs in various preclinical models. In rodent models of muscular dystrophy, the Company's Wnt7a analogs have been shown to … Continue reading →

NEW YORK, Oct. 2, 2013 /PRNewswire/ --Charley's Fund, an organization dedicated to finding a treatment and cure for Duchenne Muscular Dystrophy (Duchenne), announced its fourth annual Race Against Time fundraiser run will take place on Sunday, October 6 in Central Park. The race will be attended by more than 250 runners of all ages from across the country, with the goal of raising money to support research in Duchenne, the most common fatal genetic disorder to affect children around the world. (Logo: http://photos.prnewswire.com/prnh/20131002/NY90136LOGO ) Duchenne is characterized by progressive muscle degeneration and weakness, which typically begins in patients between the ages of three and five. This disease has a 100 percent fatality rate, with most children succumbing to it in their late teens or early 20s.Sufferers born with the disease, primarily boys, are unable to produce dystrophin, a protein essential for muscle strength and function. Finding a cure for Duchenne is truly a Race Against Time. Charley Seckler, for whom Charley's Fund is named, was diagnosed with Duchenne in 2004.After hearing Charley's diagnosis, his parents, Tracy and Benjamin, immediately set their sights on supporting the search for a cure.Through Charley's Fund, they have contributed more than $25 million dollars to … Continue reading →

Sarepta Therapeutics Inc.s ('>SRPT) shares are slowly climbing following the announcement of encouraging additional data from a phase IIb open label extension study (Study 202) on eteplirsen for the treatment of patients suffering from Duchenne muscular dystrophy (DMD). The study had met its primary endpoint of increased novel dystrophin at week 48 these results were presented earlier. 96 week data showed continued stability of walking ability in eteplirsen-treated patients. Post 96 weeks of treatment, patients taking 30 mg/kg and 50 mg/kg doses of eteplirsen experienced less than a 5% decline from baseline in walking ability as measured by the 6-minute walk test (6MWT). A statistically significant treatment benefit of 70.8 meters was observed as compared to placebo. According to information provided by Sarepta, roughly one in every 3,500 boys globally is affected by DMD. Sarepta intends to file a New Drug Application (:NDA) for eteplirsen in the first half of 2014 with the U.S. Food and Drug Administration (:FDA).The decision to file the NDA was based on productive interactions with the FDA including a meeting with the agency in July. We note that several other companies including Prosensa (RNA) are developing therapies targeting the DMD market. However, a few days … Continue reading →

Sarepta Therapeutics Inc. 's ( ">SRPT ) shares are slowly climbing following the announcement of encouraging additional data from a phase IIb open label extension study (Study 202) on eteplirsen for the treatment of patients suffering from Duchenne muscular dystrophy (DMD). The study had met its primary endpoint of increased novel dystrophin at week 48 - these results were presented earlier. 96 week data showed continued stability of walking ability in eteplirsen-treated patients. Post 96 weeks of treatment, patients taking 30 mg/kg and 50 mg/kg doses of eteplirsen experienced less than a 5% decline from baseline in walking ability as measured by the 6-minute walk test (6MWT). A statistically significant treatment benefit of 70.8 meters was observed as compared to placebo. According to information provided by Sarepta, roughly one in every 3,500 boys globally is affected by DMD. Sarepta intends to file a New Drug Application (NDA) for eteplirsen in the first half of 2014 with the U.S. Food and Drug Administration (FDA).The decision to file the NDA was based on productive interactions with the FDA including a meeting with the agency in July. We note that several other companies including Prosensa ( RNA ) are developing therapies targeting the … Continue reading →

CAMBRIDGE, MA--(Marketwired - Sep 26, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 96 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through nearly two years showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at Week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes. After 96 weeks, patients in the 30 mg/kg and 50 mg/kg eteplirsen cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) experienced less than a 5 percent decline (17.5 meters) from baseline in walking ability. A statistically significant treatment benefit of 70.8 meters (p 0.001) was observed for the mITT population compared with the placebo/delayed-treatment cohort (n=4), which initiated treatment at Week 25 following 24 weeks of placebo. After experiencing a substantial decline earlier in the study, the placebo/delayed-treatment cohort also demonstrated stabilization in walking ability from Week 36 through … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne announced today the launch of CureDuchenne Ventures, a new initiative to identify and develop a robust pipeline of therapies to treat Duchenne, the most common and lethal form of muscular dystrophy. This drug discovery and development initiative is based on CureDuchennes successful venture philanthropy model, in which the organization provides early stage funding to biotechnology, pharmaceutical companies and academic investigators to help develop breakthrough therapies to treat Duchenne. Were launching CureDuchenne Ventures at an exciting time in Duchenne research, said Debra Miller, CEO and Founder of CureDuchenne. Duchenne is a progressive disease and currently there is no treatment or cure, so our goal is to accelerate research that could benefit this generation of Duchenne boys. CureDuchenne Ventures will focus on venture philanthropists, foundations, and donors with an interest in scientific research for rare diseases, such as Duchenne. The first research project to be funded through CureDuchenne Ventures is a collaboration with biopharmaceutical company, Lexicon Pharmaceuticals (Woodlands, TX). Lexicons experimental drug, LX2931, which is in clinical development as a potential treatment of rheumatoid arthritis and autoimmune disease, is currently being evaluated for its potential to initiate muscle regeneration in studies with the mdx mouse model of … Continue reading →

Experimental treatment for muscular dystrophy Treatment uses exon skipping to counteract genetic errors causing muscular dystrophy THE NEW YORK TIMES September 24, 2013 Terri Ellsworth is convinced that her 12-year-old son Billy, who has Duchenne muscular dystrophy, is being helped by an experimental drug that counteracts the genetic mutation causing his disease. His ability to walk has not deteriorated in the two years he has been on the drug, whereas many boys with the disease would be in wheelchairs by his age. Billy opened a Gatorade bottle by himself recently, beaming from ear to ear. He even took off down an uneven dirt path without falling. He never would have done that, ever, said Ellsworth, 55, a kitchen and bath designer from Coraopolis, Pa., outside Pittsburgh. Without this drug, he would not be walking today. Such anecdotal reports, and data from small clinical trials, have raised hopes that a new genetic technique called exon skipping may slow the progression of Duchenne muscular dystrophy, finally yielding a treatment for which parents have prayed for decades. Scientists say the technique or related ones might also point the way to treatments for other inherited diseases, including Huntingtons. The idea behind exon skipping is … Continue reading →

LONDON, Sept 20 (Reuters) - An experimental drug for Duchenne muscular dystrophy from GlaxoSmithKline and Prosensa failed to meet its goal in a late-stage clinical trial, sending shares in Prosensa tumbling 75 percent in pre-market trade. The drug, drisapersen, did not show a statistically significant improvement in the distance that patients could walk in six minutes compared to placebo in the Phase III test, the companies said on Friday. The setback also unnerved investors in Sarepta Therapeutics , which is developing a rival treatment for the rare muscle-wasting disorder, and its shares fell 8 percent in pre-market Nasdaq dealings. GSK and Dutch-based Prosensa had previously been seen as ahead of the pack in making a drug for the untreatable condition, although data released on GSK's website last month had already indicated a substantial number of patients treated with drisapersen might not be getting a meaningful drug effect. The news is a fresh late-stage pipeline blow for GSK - Britain's biggest drugmaker - which reported on Sept. 5 that an experimental cancer vaccine failed to help melanoma patients. Up until now, 2013 has been a strong year for GSK research, with new drug approvals in HIV, cancer and respiratory disease. But … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--While we are disappointed by the Phase III trial results for drisapersen, we hold out hope that they will provide critical information to the research community that can be applied in future studies, including those by GlaxoSmithKline and Prosensa, said Debra Miller, CEO and Founder, CureDuchenne. We are fully confident in the exon skipping technology as a viable platform to develop a treatment for Duchenne, and Duchenne families should not give up hope. We are fully confident in the exon skipping technology as a viable platform to develop a treatment for Duchenne, and Duchenne families should not give up hope. In addition to its support of Glaxo and Prosensa, CureDuchenne is working with multiple companies to develop a robust pipeline of potential drugs to treat Duchenne. CureDuchenne also provided early support to Sarepta, another company in trials for its exon skipping drug, eteplirsen. Currently, there is no treatment for Duchenne, a progressive muscle-wasting disease and the most common and lethal form of muscular dystrophy. Duchenne impacts one in every 3,500 boys; nearly 20,000 boys are living with the disease in the United States. Boys with Duchenne are usually diagnosed before the age of 5 and are in … Continue reading →