FDA Requests More Research on Sarepta’s MD Novel Drug Before Approval

Biopharmaceutical company Sarepta Therapeutics, which specializes in developing therapies for rare and infectious diseases, has been waiting for approval from the U.S. Food and Drug Administration (FDA) for its new drug treatment for muscular dystrophy. However, the FDA has decided to delay the decision, and has asked the company for more research. The FDA…

A group of investigators from the Massachusetts Institute of Technology (MIT) recently joined forces with the Myotonic Dystrophy Foundation (MDF) and Parent Project Muscular Dystrophy (PPMD) to host the second annual “Strength, Science, & Stories of Inspiration” event, which took place last Friday, November 7 at the MIT Museum in…

Almost $800,000 in funding was recently awarded to Murdoch University researchers from the Centre for Comparative Genomics (CCG) to develop genetic drugs to treat rare diseases. The money comes from the National Health and Medical Research Council (NHMRC) and will allow for the expansion of successful research into Duchenne Muscular Dystrophy…

Investigators from the Helmholtz Zentrum München and Ludwig-Maximilians-Universität München in Germany are organizing a crowd sourcing exercise as a way of determining new algorithms that they expect will be able to accelerate their research into treatment for amyotrophic lateral sclerosis (ALS). The challenge itself, as well as the most effective algorithms found, were recently published…

A group of researchers from Richland, Washington-based Pacific Northwest National Laboratory recently conducted a study that made use of an ultrasound technique that could be synced with smartphones to allow muscular dystrophy patients to monitor their response to medication. Michael S. Hughes is the lead investigator on the research…

The Italian Government recently approved the protein restoration therapy Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD), and included it in its medication list for reimbursement by the Italian National Health System. The oral drug treatment, developed by biopharmaceutical company PTC Therapeutics, will now be available in…

A new study entitled “Identifying diagnostic DNA methylation profiles for facioscapulohumeral muscular dystrophy in blood and saliva using bisulfite sequencing” published in the Clinical Epigenetics journal designed an assay that identifies the epigenetic signature for both types of Facioscapulohumeral muscular dystrophy from a multiple collections of…

The results from Milo Biotechnology’s Follistatin Gene Therapy were published in the Molecular Therapy journal showing therapy efficacy in increasing muscle strength and function in Becker’s muscular dystrophy patients. The study is entitled “A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.” The study followed…

A novel RNA-based drug from specialized biotech company, Sarepta Therapeutics, Inc., is experiencing a delay in its New Drug Application (NDA) from the US Food and Drug Administration’s (FDA), due to the FDA’s request for specific, additional data requirements from the company. The drug, eteplirsen, is one of the company’s leading pipeline…

PTC Therapeutics, Inc. announced the results of a Phase 2b clinical trial entitled “Ataluren treatment of patients with nonsense mutation dystrophinopathy” in the October issue of Muscle & Nerve, demonstrating that Translarna™ (ataluren) improved clinical outcomes of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) in males five…