GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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CRISPR breakthrough trial aims to cure kids of lifelong blood disease
⭐️ CURE ⭐️ OngoingThis study is testing a one-time gene therapy called CTX001 in children with severe transfusion-dependent beta-thalassemia. Doctors use CRISPR gene-editing technology to modify a patient's own blood stem cells, then infuse them back, aiming to eliminate the need for lifelong bloo…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 23, 2026 15:18 UTC
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CRISPR gene therapy patients monitored for years in groundbreaking study
Disease control ENROLLING_BY_INVITATIONThis study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monito…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Mar 27, 2026 12:41 UTC
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New hope for duchenne: major trial tests cell therapy to preserve muscle function
Disease control OngoingThis study is testing whether a cell therapy called deramiocel can slow the loss of muscle function in boys and young men with Duchenne muscular dystrophy (DMD). About 106 participants will receive either the therapy or a placebo via IV infusion every three months for a year, wit…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC