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 muscular dystrophy
Disease control CompletedThis study tested a drug called ataluren in 360 boys (age 5 and older) with Duchenne muscular dystrophy caused by a specific genetic mistake called a nonsense mutation. The goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participa…
Matched conditions: GENETIC DISEASES, X-LINKED
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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Diabetes drug shows promise for fragile x symptoms in new trial
Symptom relief CompletedThis study tested if metformin, a common diabetes medicine, can help improve language, behavior, and weight control in people with Fragile X syndrome. 125 participants aged 6 to 35 were randomly assigned to receive metformin or a placebo for 4 months. The main goal was to see if …
Matched conditions: GENETIC DISEASES, X-LINKED
Phase: PHASE2 • Sponsor: University of Alberta • Aim: Symptom relief
Last updated May 13, 2026 16:01 UTC