New study aims to make duchenne gene therapy safer for kids
Disease control
Not yet recruiting
This study tests whether adding sirolimus (a drug that calms the immune system) to ELEVIDYS gene therapy can prevent serious liver injury in boys with Duchenne muscular dystrophy. About 20 participants will receive the treatment and be monitored for side effects. The goal is to m…
Phase: PHASE4 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC