MUSCULAR DYSTROPHY, DUCHENNE
Clinical trials for MUSCULAR DYSTROPHY, DUCHENNE explained in plain language.
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Heart cell therapy shows promise for duchenne MD in major trial
Disease control OngoingThis Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to …
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Phase: PHASE3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC
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Higher dose of muscular dystrophy drug shows promise in new trial
Disease control OngoingThis Phase 3 trial tests whether higher doses of eteplirsen (Exondys 51) can safely improve muscle function in boys with Duchenne muscular dystrophy whose genetic mutation allows exon 51 skipping. About 160 ambulatory boys will receive one of three doses (30, 100, or 200 mg/kg) t…
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Phase: PHASE3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC