GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
Never miss a new study
Get alerted when new GENETIC DISEASES, INBORN trials appear
Sign up with your email to follow new studies for GENETIC DISEASES, INBORN, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
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 Jun 22, 2026 12:03 UTC
-
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 22, 2026 11:56 UTC
-
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