MUSCULAR DYSTROPHIES
Clinical trials for MUSCULAR DYSTROPHIES explained in plain language.
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New hope for FSHD: experimental drug AOC 1020 completes early testing
Disease control CompletedThis study tested a new medicine called AOC 1020 in 90 adults with FSHD, a genetic muscle-weakening disease. The goal was to check if the drug is safe and to measure how it moves through the body. Participants received either the drug or a placebo by IV, and researchers looked fo…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE1, PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New drug shows promise for boys with rare form of muscular dystrophy
Disease control CompletedThis study tested a drug called ataluren in 360 boys (age 5 and older) with Duchenne muscular dystrophy caused by a specific genetic mistake called a nonsense mutation. The goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participa…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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Experimental DM1 drug shows promise in long-term safety trial
Disease control CompletedThis study looked at the long-term safety of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 people who completed an earlier study received multiple doses of the drug by IV. Researchers tracked side effects and measured drug lev…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 07, 2026 18:42 UTC
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Soft exoskeleton shows promise for muscle disorder patients
Symptom relief CompletedThis study tested a powered soft exoskeleton (MyoSuit) in 32 adults with various muscle disorders to see if it is safe and helps with walking. Participants performed walking tests with and without the device. The main goal was to check for side effects and measure any improvement…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: NA • Sponsor: Institut de Myologie, France • Aim: Symptom relief
Last updated May 07, 2026 18:40 UTC
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Key study maps muscle decline in rare dystrophy to pave way for new therapies
Knowledge-focused CompletedThis study involved 116 people with Limb Girdle Muscular Dystrophy (LGMD), a group of rare muscle-weakening diseases. Researchers measured how well participants could walk, use their arms, and breathe over time. The goal was to identify the best ways to measure disease progressio…
Matched conditions: MUSCULAR DYSTROPHIES
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated May 17, 2026 14:00 UTC
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Robot leg brace shows promise for muscle disease patients
Knowledge-focused CompletedThis study tested the safety and immediate effects of a powered leg exoskeleton (Keeogo) in 50 adults with various muscle-weakening disorders like muscular dystrophy. Participants used the device to perform walking tasks while researchers monitored for side effects and measured c…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: NA • Sponsor: Institut de Myologie, France • Aim: Knowledge-focused
Last updated May 17, 2026 13:56 UTC
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Scientists probe immune System's role in rare muscle disease
Knowledge-focused CompletedThis study looked at whether inflammation plays a role in type 1 facioscapulohumeral muscular dystrophy (FSHD1), a genetic muscle-weakening disease. Researchers measured levels of 29 inflammatory proteins in the blood of 20 adults with FSHD1 and compared them to healthy controls.…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated May 13, 2026 15:58 UTC