GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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Cell therapy shows promise in slowing duchenne muscular dystrophy
Disease control OngoingThis study tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy (DMD), a genetic disease that weakens muscles over time. Participants receive either deramiocel or a placebo every 3 months for a year, then all can receive der…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated May 26, 2026 11:18 UTC
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CRISPR gene therapy: a lifelong watch begins for blood disease patients
Disease control ENROLLING_BY_INVITATIONThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who already received an experimental CRISPR gene therapy called CTX001. Researchers will track participants for years to check for long-term side effects, new cancers, and how well the treatme…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 11:08 UTC
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CRISPR gene therapy may free kids with thalassemia from lifelong transfusions
Disease control OngoingThis study tests a single dose of CTX001, a gene therapy made from the patient's own blood stem cells edited with CRISPR, in 16 children with transfusion-dependent beta-thalassemia. The goal is to see if it can help them produce enough healthy red blood cells to stop needing regu…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 22, 2026 14:01 UTC