MUSCULAR DYSTROPHIES
Clinical trials for MUSCULAR DYSTROPHIES explained in plain language.
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Extended trial tests promising drug for genetic muscle disease
Disease control CompletedThis study continued testing an experimental drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. It followed 37 participants from a previous trial to check long-term safety and see if the drug continues to work when given every 8 weeks…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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Hope for slowing muscle loss in duchenne muscular dystrophy
Disease control CompletedThis study tested whether a drug called ataluren could slow the progression of muscle weakness in boys with a specific genetic form of Duchenne muscular dystrophy. For 72 weeks, 360 participants received either ataluren or a placebo, followed by another 72 weeks where everyone re…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 16, 2026 15:25 UTC
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Landmark study maps the course of a debilitating muscle disease
Knowledge-focused CompletedThis study aimed to define the best ways to measure how Limb Girdle Muscular Dystrophy (LGMD) changes over time. It followed 116 people with specific genetic forms of LGMD, tracking their mobility, strength, and daily function. The goal was to create a clear roadmap of the diseas…
Matched conditions: MUSCULAR DYSTROPHIES
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:35 UTC
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Scientists search for inflammation clues in rare muscle disease
Knowledge-focused CompletedThis completed pilot study aimed to understand how the immune system might contribute to facioscapulumeral muscular dystrophy type 1 (FSHD1), a genetic muscle-wasting disease. Researchers compared blood inflammation markers in 20 adults with FSHD1 to healthy people to see if spec…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:38 UTC