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 facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. The goal was to check if the drug is safe and how the body processes it. Participants received either the drug or a p…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE1, PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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New drug aims to help boys with duchenne walk longer
Disease control CompletedThis study tested a drug called ataluren in 360 boys with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). The main goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participants were also taking…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease
Disease control CompletedThis study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infu…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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Robotic leg device shows promise for helping muscle disease patients walk
Symptom relief CompletedThis study tested a powered leg exoskeleton (Keeogo) in 50 people with various muscle disorders to see if it is safe and helps them walk better. Participants performed walking tests with and without the device. The goal was to see if the device could improve walking distance and …
Matched conditions: MUSCULAR DYSTROPHIES
Phase: NA • Sponsor: Institut de Myologie, France • Aim: Symptom relief
Last updated Jun 27, 2026 12:08 UTC
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Robotic exoskeleton shows promise for muscle disease patients
Symptom relief CompletedThis study tested a wearable robotic suit called MyoSuit that assists knee and hip movement in people with various muscle disorders. 32 participants used the device to perform walking tests, and researchers checked for safety and any immediate improvements in walking ability. The…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: NA • Sponsor: Institut de Myologie, France • Aim: Symptom relief
Last updated Jun 27, 2026 12:08 UTC
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Inflammation may be key in rare muscle disease
Knowledge-focused CompletedThis pilot study examined whether inflammation, measured by cytokines in the blood, plays a role in type 1 facioscapulohumeral muscular dystrophy (FSHD1). Researchers compared 20 FSHD1 patients with healthy controls to see if certain inflammatory markers are higher in the disease…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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New study maps key tests for LGMD to speed up drug development
Knowledge-focused CompletedThis study involved 116 people with Limb Girdle Muscular Dystrophy (LGMD), a group of rare muscle-weakening disorders. Researchers measured how well participants could walk, move their arms, and breathe, and asked about their daily activities and overall health. The goal was to i…
Matched conditions: MUSCULAR DYSTROPHIES
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC