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Audentes Therapeutics And Genethon Announce Agreement To Develop Treatment For Severe Genetic Disease X-Linked Myotubular Myopathy


2/5/2014 9:00:29 AM

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SAN FRANCISCO--(BUSINESS WIRE)--Audentes Therapeutics, Inc., a biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, and Genethon, a non-profit organization dedicated to the research and development of biotherapies for orphan genetic diseases, announce that they have entered into an agreement to develop AT001 for the treatment of X-Linked Myotubular Myopathy (XLMTM), a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. AT001 is a novel drug candidate based on adeno-associated virus (AAV) gene therapy technology.

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