MUSCULAR DYSTROPHY, DUCHENNE
Clinical trials for MUSCULAR DYSTROPHY, DUCHENNE explained in plain language.
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5-Year watch: how does a duchenne drug perform in everyday life?
Disease control CompletedThis study monitored the safety and real-world effectiveness of the drug Translarna (ataluren) for people with Duchenne Muscular Dystrophy. It followed 316 patients across Europe for at least five years as they received the drug as part of their normal medical care. The goal was …
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 31, 2026 12:10 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 DYSTROPHY, DUCHENNE
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 16, 2026 15:25 UTC