MUSCULAR DYSTROPHY, DUCHENNE
Clinical trials for MUSCULAR DYSTROPHY, DUCHENNE explained in plain language.
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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 DYSTROPHY, DUCHENNE
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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Can tracking muscle changes unlock better duchenne treatments?
Knowledge-focused CompletedThis study follows boys with Duchenne muscular dystrophy who have small mutations in their genes, a group that is less understood than others. Over one year, researchers will use muscle MRI, genetic tests, and motor and respiratory assessments to see how the disease progresses. T…
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS • Aim: Knowledge-focused
Last updated Aug 05, 2026 00:00 UTC
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Real-World data on duchenne drug translarna collected in large safety study
Knowledge-focused CompletedThis study followed 316 people with Duchenne muscular dystrophy who were taking Translarna (ataluren) as part of their normal medical care. The goal was to monitor side effects and see how well the drug works in everyday use. Researchers also checked if doctors and patients follo…
Matched conditions: MUSCULAR DYSTROPHY, DUCHENNE
Sponsor: PTC Therapeutics • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:08 UTC