GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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New hope for duchenne: drug targets genetic glitch to slow muscle decline
Disease control CompletedThis study tested a drug called ataluren in 360 boys (age 5 and older) with Duchenne muscular dystrophy caused by a 'nonsense mutation' – a specific genetic typo. The goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participants we…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 17, 2026 04:54 UTC
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New drug shows promise for muscle disease in extended trial
Disease control CompletedThis study looked at the long-term safety of a drug called AOC 1001 for adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 people who completed an earlier study received multiple doses of the drug by IV. The main goal was to check for side effects, and rese…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 15, 2026 18:57 UTC
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CRISPR gene editing offers hope for thalassemia patients to ditch transfusions
Disease control CompletedThis study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long th…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 12, 2026 12:05 UTC