Genetic diseases, inborn
Clinical trials for Genetic diseases, inborn explained in plain language.
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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 2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Sep 13, 2026 00:00 UTC
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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 3 • 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 2/3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Can a detailed fetal gene test guide pregnancy care without adding stress?
Knowledge-focused CompletedResearchers are studying how prenatal exome sequencing, a detailed genetic test of fetal DNA, influences medical decisions and parental wellbeing in pregnancies where an anomaly has been detected. The study will compare different ways of analyzing the test results, including defi…
Matched conditions: GENETIC DISEASES, INBORN
Sponsor: Leiden University Medical Center • Aim: Knowledge-focused
Last updated Sep 02, 2026 00:00 UTC