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 →