Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy Based On Functional Improvements

The company presented encouraging data on functional assessments of four boys treated with a micro-dystrophin gene therapy at the World Muscle Society, furthering momentum around the program.

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Sarepta Therapeutics Inc. already presented positive data in June for its micro-dystrophin gene therapy in three boys with Duchenne Muscular Dystrophy, showing positive results on gene expression and biomarkers, but the company released the first data from functional assessments Oct. 4 at the World Muscle Society Congress.

The data were encouraging across endpoints like the North Star Ambulatory Assessment (NSAA) scale, time to rise, four stairs up...

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