GENETIC DISEASES, X-LINKED
Clinical trials for GENETIC DISEASES, X-LINKED 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: GENETIC DISEASES, X-LINKED
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 22, 2026 13:54 UTC
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Diabetes drug shows promise for fragile x symptoms in new trial
Symptom relief CompletedThis study tested whether metformin, a common diabetes medicine, can help improve language, behavior, and weight problems in people with fragile X syndrome. 125 people aged 6 to 35 took either metformin or a placebo for 4 months. Researchers measured changes in spoken language an…
Matched conditions: GENETIC DISEASES, X-LINKED
Phase: PHASE2 • Sponsor: University of Alberta • Aim: Symptom relief
Last updated May 20, 2026 11:57 UTC