GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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CRISPR gene editing frees thalassemia patients from lifelong transfusions
Disease control CompletedThis study tested a one-time treatment using CRISPR gene editing to fix a patient's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the modified cells after a short course of chemotherapy. …
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 17, 2026 03:31 UTC
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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, INBORN
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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Experimental DM1 drug shows promise in long-term safety trial
Disease control CompletedThis study looked at the long-term safety of a drug called AOC 1001 in 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. Researchers tracked side effects and measured drug lev…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 07, 2026 18:42 UTC