Sarepta begins ENDEAVOR Cohort 8 trial of ELEVIDYS with sirolimus in 25 non-ambulant Duchenne patients

Reuters03-16 20:33

Sarepta begins ENDEAVOR Cohort 8 trial of ELEVIDYS with sirolimus in 25 non-ambulant Duchenne patients

Sarepta reported that screening and enrollment have begun in Cohort 8 of the open-label Phase 1b ENDEAVOR study evaluating ELEVIDYS in non-ambulant males with Duchenne muscular dystrophy. The cohort plans to enroll about 25 U.S. participants who will receive prophylactic sirolimus starting 14 days before infusion and continuing for 12 weeks afterward. The regimen is intended to assess whether sirolimus can reduce acute liver injury risk associated with AAV gene therapy in non-ambulant patients. Primary endpoints include the incidence of acute liver injury and ELEVIDYS micro-dystrophin expression at 12 weeks. No study results were reported in the announcement.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Sarepta Therapeutics Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 202603160833BIZWIRE_USPR_____20260316_BW238001) on March 16, 2026, and is solely responsible for the information contained therein.

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