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

 
• By Jessica Merrill

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.

Development Attainment Motivation Career Growth Concept. Mans Hand Reaching For Red Ladder Leading To A Blue Sky

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 and 100m walk, suggesting the gene

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