GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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New hope for duchenne: targeted therapy now available for eligible patients
Disease control AVAILABLEThis program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …
Matched conditions: GENETIC DISEASES, INBORN
Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients
Disease control Recruiting nowThis study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or s…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New gel could help heal wounds in rare skin disease
Disease control Recruiting nowThis study tests a gel called Oleogel-S10 on skin wounds in people with inherited epidermolysis bullosa (EB), a condition that causes fragile, blistering skin. The trial includes 6 Japanese children and adults with junctional or dystrophic EB. Participants apply the gel to wounds…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC