Category Archives: Muscular Dystrophy Treatment

Featured Article Academic Journal Main Category: Muscular Dystrophy / ALS Also Included In: Rehabilitation / Physical Therapy Article Date: 05 Aug 2013 - 8:00 PDT Current ratings for: Could cola substance treat muscular dystrophy? 5 (1 votes) Researchers have discovered that a trace substance found in caramelized sugar and cola improves regeneration in mice suffering from Duchenne muscular dystrophy (DMD). Scientists from the Reyes and Ruohola Baker laboratories at the University of Washington discovered that when the sugar substance, a small molecule called 2-acetyl-4(5)-tetrahydroxybutyl imidazole (THI), was injected into mice suffering from the muscular disease, muscle regeneration was improved. The study, published in the journal Skeletal Muscle, tested mice that were missing the gene that produces dystrophin - a muscle repair protein. This is the same gene that is missing in boys suffering from DMD. Muscular dystrophy is a term for a number of hereditary, progressive degenerative muscle disorders. It affects skeletal muscles and can often affect other organ systems. DMD is the most common form of the disorder, mainly affecting boys, and onset of the disorder is usually between 3 and 5 years of age. The disorder can progress rapidly, meaning that the majority of boys are unable to … Continue reading →

By Kanak Kanti - August 2, 2013 | Tickers: GSK, RNA, SRPT | 0 Comments Kanak is a member of The Motley Fool Blog Network -- entries represent the personal opinion of the blogger and are not formally edited. Two biotech companies, Sarepta Therapeutics (NASDAQ: SRPT) and Prosensa Holdings (NASDAQ: RNA) are involved in a race to be the first to get their candidate approved for treatment of DMD, a rare recessive form of muscular dystrophy. However, what is involved is a lot more than just the financial angle. Approvals of these two drugs can potentially change the focus of future research in discovery of new drugs. Sarepta Therapeutics, a company engaged in development of RNA-based therapeutics, recently got the go-ahead from the FDA for filing a new drug application (NDA) for its candidate for treatment of Duchenne muscular dystrophy (DMD), eteplirsen, on the basis of existing studies, and will be submitting the NDA in the first half of 2014. Drisapersen of Prosensa, a Dutch biotech company, which was granted Breakthrough Designation by the FDA last month, is the other drug in the race. The global pharmaceutical giant GlaxoSmithKline (NYSE: GSK) holds the license for developing and commercializing drisapersen. A … Continue reading →

Washington, Aug 02 : A burnt sugar derivative, when purified and given in appropriate doses, helps in improving muscle regeneration in a mouse model of Duchenne muscular dystrophy. The researchers from the University of Washington explained that the mice in their study, like boys with the gender-linked inherited disorder, are missing the gene that produces dystrophin, a muscle-repair protein. Professor of biochemistry and associate director of the Institute for Stem Cell and Regenerative Medicine, Hannele Ruohola-Baker's lab originally identified the sphingosine 1-phosphate (S1P) pathway as a critical player in ameliorating muscular dystrophy in flies. Reyes said that the multi-talented, bioactive lipid is essential in turning stem cells into specific types of cells, in regenerating damaged tissue, and in inhibiting cell death. Without cell receptors for sphingosine 1-phosphate, an embryo would fail to develop. Other scientists had observed that levels of sphingosine 1-phosphate are lower in the muscles of mice with the muscular dystrophy mutation, and that certain cell repair pathways involving this signal are impaired. By using insect activity monitors, the scientists assessed the effects of drug and gene therapy candidates on the flies' ability to move. This screening tool led to the discovery that a small molecule with a … Continue reading →

Aug. 1, 2013 A trace substance in caramelized sugar, when purified and given in appropriate doses, improves muscle regeneration in a mouse model of Duchenne muscular dystrophy. The findings are published today (Aug. 1) in the journal Skeletal Muscle. Morayma Reyes, professor of pathology and laboratory medicine, and Hannele Ruohola-Baker, professor of biochemistry and associate director of the Institute for Stem Cell and Regenerative Medicine, headed the University of Washington team that made the discovery. The first authors of the paper were Nicholas Ieronimakis, UW Department of Pathology; and Mario Pantoja, UW Department of Biochemistry. They explained that the mice in their study, like boys with the gender-linked inherited disorder, are missing the gene that produces dystrophin, a muscle-repair protein. Neither the mice nor the affected boys can replace enough of their routinely lost muscle cells. In people, muscle weakness begins when the boys are toddlers, and progresses until, as teens, they can no longer walk unaided. During early adulthood, their heart and respiratory muscles weaken. Even with ventilators to assist breathing, death usually ensues before age 30. No cure or satisfactory treatment is available. Prednisone drugs relieve some symptoms, but at the cost of severe side effects. The disabling, … Continue reading →

Leiden, The Netherlands, Aug. 1, 2013 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that it has been awarded a prestigious Framework Programme 7 (FP7) research grant from the European Union to support the ongoing clinical study of the Company's third novel development candidate, PRO045, for the treatment of Duchenne muscular dystrophy (DMD). The project, titled "Consortium for Products Across Europe in Duchenne Muscular Dystrophy" (SCOPE-DMD) is expected to run for three years. Prosensa is part of a high-quality, pan-European consortium that has been awarded the grant of approximately EUR 6 million to evaluate its exon 45-skipping drug candidate, PRO045, in an innovatively designed clinical proof-of-concept study in boys with DMD. The consortium includes leading expert centers in DMD such as Newcastle University in the United Kingdom, the coordinating partner within the project, the Institute of Myology in France and Leiden University Medical Center in the Netherlands, as well as an industrial partner, BioSpring in Germany. The combined experience and expertise of this consortium will help advance the development of PRO045 which entered clinical trials earlier this year. "We are delighted with this funding, … Continue reading →

PLEASANTON, Calif., July 31, 2013 /PRNewswire/ --Safeway Inc., (SWY) and The Safeway Foundation are teaming up with The Muscular Dystrophy Association (MDA) for an annual fundraising campaign to support individuals and families affected by muscle disease. (Logo: http://photos.prnewswire.com/prnh/20130625/SF38175LOGO) From July 31 through September 2, all Safeway stores including Vons, Pavilions, Randalls, Tom Thumb, Dominick's and Carrs will participate in the companywide campaign, giving customers the opportunity to contribute at the checkout register and help raise funds for MDA's programs and services. "Children and adults served by MDA will benefit directly from every donation made at our stores," said Larree Renda, Safeway Executive Vice President and Chair of The Safeway Foundation. "The donations will contribute to MDA's worldwide research program, its national network of 200 medical clinics, and will help send thousands of children to a weeklong MDA summer camp at nearly 80 locations throughout the United States." Over the years, individual Safeway stores and operating divisions have collected donations for the organization through local grassroots fundraising efforts. The company and The Safeway Foundation have raised more than $68.7 million for vital services and resources for those affected by muscular dystrophy and related muscle diseases. "We're grateful to have the support … Continue reading →

Your Invitation to ROCK THE CURE with Kaye/Bassman Foundation - Oct 18th at Concrete Cowboy Learn more at http://www.kbicfoundation.org. You are invited to help support our 12th Annual Charity Fundraiser, the Kaye/Bassman Foundation's "Rock the Cure... By: Kaye Bassman … Continue reading →

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CAMBRIDGE, MA--(Marketwired - Jul 24, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced it plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2014 for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).Eteplirsen is Sarepta's lead exon-skipping compound in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51. The decision to submit an NDA for eteplirsen in 2014 is based on productive interactions with the FDA in a meeting that occurred this week.That meeting was a follow-up to the FDA's review of two recently submitted summary documents that included data on dystrophin and clinical outcomes from the existing eteplirsen studies.The FDA stated in pre-meeting comments that the Agency is "open to considering an NDA based on these data for filing."The Agency, however, requested additional information related to the methodology and verification of dystrophin quantification.Sarepta believes the requests from the Agency can be addressed and incorporated into an NDA submission in the first half of 2014. "We are encouraged by the feedback from the FDA and believe that data … Continue reading →

Sarepta Therapeutics (SRPT) Inc. plummeted the most in 14 months on investor concerns that U.S. regulators wont consider the companys treatment for Duchenne muscular dystrophy on an accelerated basis. Sarepta, with no products on the market, fell 12 percent to $40.95 at 11:11 a.m. The shares of the Cambridge, Massachusetts-based company declined as much as 17 percent, their biggest intraday drop since May 2012, after at least three analysts cut their ratings on the stock. The biotechnology company announced today it plans to seek approval of eteplirsen in the first half of 2013 after the Food and Drug Administration said it was open to a filing based on data from existing studies. The agency requested information on methodology and verification that will be incorporated into the filing, the company said in a statement today. Regulators didnt commit to considering approval of the compound on an accelerated approval basis, Sarepta said. If youre a bear, you believe that the FDA is just saying file, and the management is filing just because they can, not because they think theyre going to get approved, Robyn Karnauskas, a New York-based analyst at Deutsche Bank AG, said in a telephone interview. She reduced her rating … Continue reading →