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 duchenne muscular dystrophy
Disease control CompletedThis study tested a drug called ataluren in 360 boys (age 5 and older) with a specific genetic form of Duchenne muscular dystrophy caused by a 'nonsense mutation.' The goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. Participants conti…
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 22, 2026 13:54 UTC
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Real-World data on duchenne drug safety collected in completed study
Knowledge-focused CompletedThis study followed 316 people with Duchenne muscular dystrophy who were already taking Translarna (ataluren) 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 informa…
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Sponsor: PTC Therapeutics • Aim: Knowledge-focused
Last updated May 22, 2026 13:50 UTC