Day 19 of 30 Days of MD: Hurricane Prep and MD

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I am proud to say that Muscular Dystrophy News Today is running a great initiative called “30 Days of MD.” This series of stories by patients, caregivers, researchers, and others will continue for the duration of September and will end with the day now recognized as LGMD Awareness…

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Due to a lack of efficacy, Acceleron Pharma is halting the clinical development of its ACE-083 candidate for the treatment of facioscapulohumeral muscular dystrophy (FSHD) . Topline results from a Phase 2 clinical study showed that ACE-083 was well-tolerated and could significantly increase muscle mass, which was…

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Researchers have identified a new set of blood biomarkers of Duchenne muscular dystrophy (DMD) and of response to standard glucocorticoid therapy. The study, conducted by U.S. and Canadian researchers, also found that glucocorticoid treatment can result in significant changes in the levels of proteins not related to DMD, highlighting…

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A review study highlights the benefits of adding an extra protein domain —  called neuronal nitric oxide synthase (nNOS) — to microdystrophin gene therapy. nNOS is an important player in regulating blood flow to muscle cells, and the reviewed evidence suggests it protects muscle against restricted blood flow (a condition…