GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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Three-Year extension trial tests promising muscle disease treatment
Disease control CompletedThis study followed patients who completed an earlier trial to evaluate the long-term safety and effectiveness of AOC 1001, an experimental treatment for myotonic dystrophy type 1. Thirty-seven adults with this genetic muscle disease received intravenous doses every 8 weeks for u…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Apr 04, 2026 05:07 UTC
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One-Shot CRISPR treatment aims to free patients from lifelong blood transfusions
Disease control CompletedThis study tested a one-time treatment using the gene-editing tool CRISPR to try to help people with severe thalassemia. Doctors took a patient's own blood stem cells, edited a gene in the lab, and then gave them back. The main goal was to see if this single treatment could allow…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Apr 02, 2026 14:57 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