Sarepta Complained To Woodcock About Exondys 51 Review Early And Often
Executive Summary
Documents also show that Sarepta officials begged CDER director for approval on multiple occasions prior to the agency's September 2016 decision.
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Sarepta’s DMD Gene Therapy Adcomm Likely To Focus On Dystrophin As A Surrogate Endpoint
Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
Exondys Revisited? Translarna Brings Efficacy Woes Into US Panel Review
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