New Follistatin Gene Therapy Improves Function in Becker Muscular Dystrophy

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October 31, 2014↗ source

Coverage of the Phase 1/2a trial of AAV-delivered FS344 follistatin gene therapy in Becker muscular dystrophy patients. The majority of patients showed significant gains in six-minute walk distance and stair-climbing tests. Investigator Jerry Mendell called it the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy.

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