GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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Experimental cell therapy aims to slow muscle loss in duchenne MD
Disease control OngoingThis phase 3 trial tests whether a cell therapy called deramiocel (CAP-1002) can slow muscle decline in boys and young men with Duchenne muscular dystrophy (DMD). About 106 participants receive either the therapy or a placebo every 3 months for a year, then all can receive the th…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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CRISPR treatment frees kids from lifelong blood transfusions
Disease control OngoingThis study tests a one-time gene-editing treatment (CTX001) in 16 children with transfusion-dependent beta-thalassemia, a severe blood disorder. The therapy uses the child's own stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The main goal is to see if …
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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CRISPR gene therapy: a Long-Term look at safety for blood disease patients
Disease control ENROLLING_BY_INVITATIONThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). Researchers will monitor participants for years to check for side effects, new cancers, and how well the thera…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 13, 2026 16:00 UTC