MUSCULAR DYSTROPHY, DUCHENNE
Clinical trials for MUSCULAR DYSTROPHY, DUCHENNE explained in plain language.
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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 DYSTROPHY, DUCHENNE
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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Real-World data on duchenne drug safety collected in 316-Patient study
Knowledge-focused CompletedThis study followed 316 people with Duchenne muscular dystrophy who were already taking the drug Translarna as part of their normal care. Researchers tracked side effects and how well the drug worked in real life, not in a controlled trial. The goal was to gather safety informati…
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Sponsor: PTC Therapeutics • Aim: Knowledge-focused
Last updated Apr 30, 2026 15:50 UTC