GENETIC DISEASES, X-LINKED
Clinical trials for GENETIC DISEASES, X-LINKED explained in plain language.
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Diabetes drug tested as potential treatment for genetic disorder
Disease control CompletedThis study tested whether metformin, a common diabetes medication, could help people with Fragile X syndrome. For four months, 125 participants aged 6 to 35 took either metformin or a placebo pill. Researchers measured changes in language skills, behavior problems, and weight to …
Matched conditions: GENETIC DISEASES, X-LINKED
Phase: PHASE2 • Sponsor: University of Alberta • Aim: Disease control
Last updated Mar 23, 2026 15:14 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: GENETIC DISEASES, X-LINKED
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 16, 2026 15:25 UTC