GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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Genom att skicka in godkänner du våra Användarvillkor
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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 and check for any side effects. Part…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated May 26, 2026 06:06 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 CRISPR-based gene therapy (CTX001) in 26 adults with transfusion-dependent beta-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, which are edited to produce more fetal hemoglobin, potentially red…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 05:58 UTC
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New hope for DMD: experimental drug opens to more patients
Disease control AVAILABLEThis program provides access to an investigational drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. It is for eligible patients aged 6 and older living in the US. The goal is to offer treatment while gath…
Matched conditions: GENETIC DISEASES, INBORN
Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 26, 2026 05:51 UTC