Genetic diseases, X-linked
Clinical trials for Genetic diseases, X-linked 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: GENETIC DISEASES, X-LINKED
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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Diabetes drug metformin tested for fragile x symptoms
Symptom relief CompletedThis study tested whether metformin, a common diabetes medication, can help improve language, behavior, and weight problems in people with fragile X syndrome. 125 participants aged 6 to 35 took either metformin or a placebo for 4 months. Researchers measured changes in language s…
Matched conditions: GENETIC DISEASES, X-LINKED
Phase 2 • Sponsor: University of Alberta • Aim: Symptom relief
Last updated Jun 27, 2026 08:09 UTC