The Muscular Dystrophy Association (MDA) has asked the medical data services company IQVIA to expand its disease registry into a hub of information on seven neuromuscular conditions, including spinal muscular dystrophy (SMA). ... Read more
A Phase 3 clinical trial to evaluating the safety and efficacy of an investigational gene replacement therapy by AveXis is now recruiting infants with spinal muscular atrophy type 1.
The trial ... Read more
Nine-year-old Reagan Imhoff has been chosen as the 2015 National Goodwill Ambassador, after spending 2014 traveling across the country to give a face and a voice to the Muscular Dystrophy Association (MDA). ... Read more
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