MUSCULAR DISEASE
Clinical trials for MUSCULAR DISEASE 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 DISEASE
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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Massive study seeks answers for rare inherited nerve diseases
Knowledge-focused Recruiting nowThis study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…
Matched conditions: MUSCULAR DISEASE
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Knowledge-focused
Last updated Jul 10, 2026 00:00 UTC