GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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One-Shot CRISPR cure trial for devastating blood diseases
⭐️ CURE ⭐️ Recruiting nowThis study is testing a single treatment using the patient's own stem cells that have been genetically modified with CRISPR technology. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in beta-thalassemia patients and stop painful…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 25, 2026 14:08 UTC
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New gel offers hope for healing fragile skin in rare genetic condition
Disease control Recruiting nowThis study is testing whether Oleogel-S10 gel helps close wounds in people with two types of epidermolysis bullosa (EB), a rare genetic condition that causes fragile, blistering skin. Japanese children and adults with confirmed EB will apply the gel to their wounds for 45 days wh…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated Mar 25, 2026 14:07 UTC
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New hope for boys with devastating muscle disease
Disease control AVAILABLEThis program provides access to an investigational drug called AOC 1044 for eligible patients with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation (amenable to exon 44 skipping). It is for patients aged 6 and older who are living in the US, including those …
Matched conditions: GENETIC DISEASES, INBORN
Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Mar 10, 2026 12:53 UTC