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Sarepta's DMD Gene Therapy: Durable Responses After Two Years

After A Single Infusion

 
• By John Davis

Durable responses have continued to be seen two years after administration of Sarepta’s candidate gene therapy to four boys with Duchenne muscular dystrophy.    

DNA

Sarepta Therapeutics, Inc.’s investigational gene therapy SRP-9001 is still associated with functional improvements in four patients with Duchenne muscular dystrophy two years after administration of the gene in the open-label 101 study, and the company says it is looking towards results from the double-blind 102 study, expected in early 2021. 

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