NEUROMUSCULAR DISEASES (NMD)
Clinical trials for NEUROMUSCULAR DISEASES (NMD) explained in plain language.
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Promising new treatment for duchenne muscular dystrophy enters final testing phase
Disease control Not yet recruitingThis study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The drug aims to help muscles work better by skipping a faulty part of the gene. About 70 boys aged 7 to 16 will receive either the drug or …
Matched conditions: NEUROMUSCULAR DISEASES (NMD)
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 18, 2026 12:02 UTC
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New study to settle debate: surgery or therapy for CMT walking problems?
Knowledge-focused Not yet recruitingThis study looks at 200 people with Charcot-Marie-Tooth disease (CMT) who have foot problems that make walking hard. Researchers want to see if surgery to fix the foot followed by rehab leads to better walking than physical therapy alone. Participants will be followed for up to t…
Matched conditions: NEUROMUSCULAR DISEASES (NMD)
Sponsor: Peking University Third Hospital • Aim: Knowledge-focused
Last updated May 26, 2026 03:09 UTC