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…
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 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…
The American Academy of Neurology (AAN) and the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) recently released new guidelines on the evaluation of MD patients by physicians, recommending that professionals should review all facts regarding symptoms, family history, ethnicity, physical exams, and lab tests in order to determine the…
New discoveries in the laboratory are leading to exciting, new possibilities for developing the next generation of Muscular dystrophy therapies. Recently, researchers found that by targeting muscle repair, rather than that the genetic defect that is the underlying cause for muscular dystrophy, they may be able to treat the…
Clinical stage biopharmaceutical company Akashi Therapeutics recently acquired the rights for a peptide therapy to regulate the calcium levels in the muscles, a symptom experienced by Duchenne muscular dystrophy (DMD) patients that causes loss of function and other pathologies. The treatment, called GsMTx-4, was developed by the Buffalo start-up Tonus Therapeutics, based…
Beverly Hills, California-based Capricor Therapeutics, Inc. has just announced plans to explore the potential of using one of their leading pipeline products indicated for heart disease, for the treatment of complications from an extremely rare muscle-wasting disease called, Duchenne Muscular Dystrophy (DMD). This specialized biotechnology company has…
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