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

tal in Columbus, Ohio, is lead investigator in Sarepta's trial of the exon-skipping drug eteplirsen. On Sarepta's behalf, Mendell will report on the 74-week results of its phase 2b eteplirsen trial in 12 boys with Duchenne MD.

Thomas Voit , medical and scientific director of the Institut de Myologie in Paris, will offer data on behalf of GSK/Prosensa from the company's phase 2b study of its own exon-skipping compound for Duchenne, drisapersen. 

Eric Hoffman , Ph.D., director of the Research Center for Genetic Medicine at Children's National Medical Center in Washington, D.C., is serving with Frank Bennett as co-chair of the 2013 conference. Hoffman, a longtime MDA research grantee, was a key investigator in the identification of the role of the dystrophin protein in Duchenne and Becker muscular dystrophies, and is currently studying asynchronous repair in Duchenne.

"This is an extremely promising moment in neuromuscular science, with more clinical trials of potential therapies under way than at any time in history," Hoffman said. "I can say with confidence that if it were not for MDA's fostering of innovative science and its sponsoring of conferences such as this one, prospects for families affected by neuromuscular diseases would be much less hopeful."

MDA's first national-scale scientific conference took place in Las Vegas in March, 2011, with 300 people attending. The biennial scientific gatherings take place in odd-numbered years, with MDA conferences emphasizing clinical modalities held in even-numbered years. 

Biopharmaceutical companies serving as sponsors of the 2013 MDA Scientific Conference are: Sanofi (platinum level); Biogen Idec and Sarepta'/>"/>

SOURCE Muscular Dystrophy Association
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