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MDA Scientific Conference To Focus On Therapy Development For Neuromuscular Diseases
Date:4/22/2013

ophic-lateral-sclerosis/overview">amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA); a report on the potential SMA therapy, ISIS-SMNRx, is on the conference agenda. 

Bennett added: "MDA is to be lauded for its leadership role in supporting and partnering with diverse players in the neuromuscular arena, each of whom brings unique wisdom and experience to the challenging task of developing effective therapies."

Therapy development for SMA, ALS, facioscapulohumeral muscular dystrophy, myotonic dystrophy, Duchenne muscular dystrophy, Becker muscular dystrophy, and other progressive and potentially life-threatening neuromuscular diseases will be under discussion.

Up-to-the-minute news to be presented at the conference also will include reports from:  

  • Massachusetts-based Sarepta Therapeutics (exon skipping, eteplirsen, Duchenne dystrophy);
  • London-headquartered GlaxoSmithKline (GSK), in collaboration with Dutch-based Prosensa (exon skipping, drisapersen, Duchenne MD);
  • San Francisco's Cytokinetics (troponin activator, tirasemtiv, amyotrophic lateral sclerosis);
  • Massachusetts-headquartered Repligen (HDAC inhibitors, Friedreich's ataxia); and
  • MDA-funded trial (lisinopril and losartan, cardiomyopathy in Duchenne MD).

Jerry Mendell , a longtime MDA research grantee and co-director of the MDA Clinic at Nationwide Children's Hospi
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SOURCE Muscular Dystrophy Association
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