Muscular dystrophies (Duchenne, Becker, myotonic dystrophy)
Clinical trials for Muscular dystrophies (Duchenne, Becker, myotonic dystrophy) explained in plain language.
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Hope for duchenne: new drug targets rare gene mutation in phase 3 trial
Disease control Recruiting nowThis study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…
Matched conditions: MUSCULAR DYSTROPHIES (DUCHENNE, BECKER, MYOTONIC DYSTROPHY)
Phase 3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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New drug could help boys with duchenne walk and move longer
Disease control Recruiting nowThis study tests a new medicine called DYNE-251 in boys aged 4 to 18 with Duchenne muscular dystrophy (DMD) who can still walk. The goal is to see if it helps them move better and slows muscle damage. Participants will receive either the drug or a placebo every 4 weeks for about …
Matched conditions: MUSCULAR DYSTROPHIES (DUCHENNE, BECKER, MYOTONIC DYSTROPHY)
Phase 3 • Sponsor: Dyne Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC