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Sarepta Begins Enrollment in Study Evaluating Safety Regimen With Duchenne Therapy
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Sarepta Begins Enrollment in Study Evaluating Safety Regimen With Duchenne Therapy
Mar 16, 2026 9:00 AM

11:27 AM EDT, 03/16/2026 (MT Newswires) -- Sarepta Therapeutics ( SRPT ) said Monday screening and enrollment have begun for cohort 8 of the phase 1b study evaluating its gene therapy Elevidys, or delandistrogene moxeparvovec-rokl.

The trial will assess prophylactic sirolimus as part of an enhanced immunosuppression regimen during treatment with Elevidysin non-ambulatory individuals with Duchenne muscular dystrophy, or DMD.

The study will examine whether administering sirolimus before and after Elevidys infusion can help reduce the risk of acute liver injury associated with adeno-associated virus gene therapy, the company said.

About 25 non-ambulatory participants in the US are expected to enroll. Primary endpoints include the incidence of acute liver injury and Elevidys micro-dystrophin expression at 12 weeks.

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