Sarepta Sinks On Mixed DMD Gene Therapy Study

Blames 'Bad Luck In Randomization Process'

Any time advantage Sarepta's Duchenne muscular dystrophy gene therapy may have had over rivals, notably Pfizer, has disappeared after SRP-9001 failed to significantly improve muscle function in a closely watched trial.

dmd
Dystrophin is the protein missing in children born with DMD • Source: Shutterstock

Shares in Sarepta Therapeutics, Inc. have slid after a keenly anticipated study showed that SRP-9001, its investigational gene therapy for Duchenne muscular dystrophy (DMD), failed to significantly improve muscle function, dealing a blow to hopes that the one-time treatment would get approval for the devastating childhood disease any time soon.

Sarepta has unveiled top-line results from the first part of the 41-patient Study 102, the first placebo-controlled trial of SRP-9001,...

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