GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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One-Shot CRISPR treatment aims to cure blood disorder, end lifelong transfusions
⭐️ CURE ⭐️ CompletedThis study tested a one-time treatment for people with severe beta-thalassemia, a genetic blood disorder that requires regular blood transfusions to survive. Doctors used CRISPR gene-editing technology to modify a patient's own blood stem cells, then gave them back as a transplan…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 25, 2026 14:09 UTC
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Extended trial tests promising drug for genetic muscle disease
Disease control CompletedThis study continued testing an experimental drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. It followed 37 participants from a previous trial to check long-term safety and see if the drug continues to work when given every 8 weeks…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:32 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, INBORN
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 16, 2026 15:25 UTC