GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN 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, INBORN
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 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 27, 2026 08:03 UTC
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Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease
Disease control CompletedThis study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infu…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC