GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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New gel shows promise for rare skin disease in japanese patients
Disease control Recruiting nowThis study tests a gel called Oleogel-S10 on skin wounds in Japanese children and adults with two types of epidermolysis bullosa (EB), a genetic condition causing fragile skin. The main goal is to see if the gel can close wounds within 45 days or reduce their size. Participants a…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated Jun 16, 2026 12:53 UTC
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CRISPR gene therapy aims to free patients from lifelong transfusions
Disease control Recruiting nowThis study tests a single dose of a person's own CRISPR-edited stem cells to treat two inherited blood disorders: transfusion-dependent beta-thalassemia and severe sickle cell disease. The goal is to help the body produce healthy red blood cells and reduce or eliminate the need f…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 15, 2026 19:07 UTC
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New hope for duchenne: experimental drug now available for eligible patients
Disease control AVAILABLEThis program offers access to an experimental drug called AOC 1044 for people with Duchenne muscular dystrophy whose genetic mutation can be treated by skipping exon 44. It is for patients aged 6 and older who live in the US. The goal is to provide treatment while gathering more …
Matched conditions: GENETIC DISEASES, INBORN
Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 15, 2026 19:06 UTC