A team of researchers from the University of Portsmouth recently found that in Duchenne muscular dystrophy, a phenomena called autophagy occurs where the damaged muscle cells destroy themselves. Dr. Chris Young discovered this mechanism. The research group led by Professor Darek Gorecki is working on a study focused on Duchenne muscular dystrophy.
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Researchers at Nationwide Children’s Hospital in Columbus, Ohio have developed a method for measurement of upper extremity movement in muscular dystrophy patients that employs interactive video game technology. The objective is an expansion of inclusion criteria for patients using wheelchairs who participate in clinical trials. Duchenne muscular dystrophy,…
California-based biotech company, Capricor Therapeutics, Inc., recently held a webinar on Wednesday, January 21, 2015 on the company’s latest research and development updates on addressing the largely unmet clinical needs of Duchenne muscular dystrophy (DMD) and related heart disease, which is known to be the most common cause of mortality…
The Kids Wish Network, an international charitable organization that grants wishes to children with life-threatening medical conditions, recently congratulated one of its celebrity Guardian Angel Wish Sponsors for being selected to win a humanitarian award. American country singer and songwriter, Martina McBride, will receive the distinguished Harry Chapin Memorial Humanitarian Award from the Music…
Marathon Pharmaceuticals, LLC, a biopharmaceutical company currently developing treatments for rare diseases, recently announced that the FDA granted the Fast Track designation for their deflazacort drug to treat Duchenne Muscular Dystrophy (DMD). In addition, the company is also developing several strategies to address neurological movement and muscular disorders. DMD is a fatal…
Non-profit organization Parent Project Muscular Dystrophy (PPMD) has nominated John Porter, PhD. as its new CEO. The former program director of the National Institute of Neurological Disorders and Stroke at National Institutes of Health (NIH) will join the organization to assist in supporting research projects working towards…
Last week in Europe, Sarepta Therapeutics announced the initiation of their first human trial for the new drug SRP-4053 for Duchenne Muscular Dystrophy (DMD). SRP-4053 has shown remarkable promise in treating this debilitating childhood disease in the laboratory, and researchers are highly optimistic of the potential outcomes of a human trial.
Based on the fact that in vitro models of human skeletal muscle cannot recapitulate human muscle functions, a team of researchers from Duke University developed human muscle tissues called “myobundles” using primary myogenic cells. The researchers observed that these muscle tissues were able to respond to electrical stimuli…
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Sarepta Therapeutics Reports results from 168 Weeks of Eteplirsen in Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc, a company focused on developing RNA-based therapeutics, recently announced their findings through Week 168 from Study 202, a phase IIb study that is evaluating the efficacy of a drug called eteplirsen in patients with Duchenne muscular dystrophy (DMD). The company’s study 201 was a randomized, double-blind,…
Last year, 2014, an inspiring 9-year-old girl from Wisconsin travelled all over the United States as one of the ambassadors of the Muscular Dystrophy Association. This year, she resumes her advocacy as a National Goodwill Ambassador, determined to strengthen awareness and support for muscular dystrophy, which…
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