Trial of SRP-9001 Gene Therapy Opens in California to Boys Age 3

Trial of SRP-9001 Gene Therapy Opens in California to Boys Age 3

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Trial of SRP-9001 Gene Therapy Opens in California to Boys Age 3

A Phase 1 trial of SRP-9001, Sarepta Therapeutics‘ investigational gene therapy, is recruiting 3-year-old boys with Duchenne muscular dystrophy (DMD) who are able to walk for a new patient group in California, according to a press release from the Muscular Dystrophy Association. This group, being enrolled at the Stanford Neuroscience Health Center study site, is an addition to the open-label ENDEAVOR trial (NCT04626674) testing the efficacy and safety of a single infusion of a commercially manufactured…

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