Duchenne muscular dystrophy
Clinical trials for Duchenne muscular dystrophy explained in plain language.
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New study aims to make duchenne gene therapy safer for kids
Disease control Not yet recruitingThis 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…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase 4 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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Can a new dosing schedule tame steroid side effects in duchenne?
Disease control Not yet recruitingThis trial tests two ways of taking the steroid prednisolone in boys aged 4-10 with Duchenne muscular dystrophy who can still walk. One group takes the drug every other day, while the other takes it for 10 days on, then 10 days off. The goal is to see which schedule better preser…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Sponsor: Muhammad Aamir Latif • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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Experimental drug KER-065 aims to slow muscle decline in duchenne muscular dystrophy
Disease control Not yet recruitingThis phase 2 trial tests an experimental drug called KER-065 in adult and pediatric males with Duchenne Muscular Dystrophy (DMD), a genetic condition that causes progressive muscle weakness. The study includes both those who can walk and those who cannot, and all participants mus…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase 2 • Sponsor: Keros Therapeutics, Inc. • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC