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Sarepta Sinks On Mixed DMD Gene Therapy Study

Blames 'Bad Luck In Randomization Process'

 
• By Kevin Grogan

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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