Muscular Dystrophies
MDA partnered with a French muscle disease association to host a symposium on advancing gene therapy for neuromuscular diseases
PTC Therapeutics' large-scale multinational trial of ataluren for nonsense-mutation Duchenne or Becker MD has opened its first site in Cincinnati, Ohio
In early 2011, clinical trials of ACE-031, a myostatin inhibitor, were halted pending resolution of safety issues; in May 2013, the developers discontinued work on the drug
ARMGO Pharma is developing a novel drug based on a new strategy for treating muscle weakness and degeneration in Duchenne MD, Becker MD and potentially other muscle disorders
FDA has asked Sarepta Therapeutics for additional information about dystrophin production and safety before it will consider accepting an application for accelerated approval of its exon-skipping drug
Weekly injections of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy resulted in a significant difference in walking distance compared to a placebo
10 boys with Duchenne muscular dystrophy continue to show stabilization of walking ability; further trial results will be announced at an MDA conference in late April
MDA-supported researchers have developed a research mouse with the same molecular abnormalities that underlie human types 1 and 2 facioscapulohumeral muscular dystrophy
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Muscular Dystrophy Association — USA
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3300 E. Sunrise Drive
Tucson, AZ 85718
(800) 572-1717
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