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.
By submitting, you agree to our Terms of use
-
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 by allowing the body to produce a shorter but still functional dyst…
Matched conditions: GENETIC DISEASES, INBORN
Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 17, 2026 02:38 UTC
-
New gel could help heal painful skin wounds in rare disease
Disease control Recruiting nowThis study tests a gel called Oleogel-S10 on skin wounds from two types of epidermolysis bullosa (EB), a rare genetic condition that makes skin very fragile. About 6 Japanese children and adults will apply the gel to their wounds at dressing changes for 45 days, with the option t…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated May 17, 2026 02:34 UTC
-
CRISPR gene editing offers hope for blood disorder patients
Disease control Recruiting nowThis study tests a single dose of a CRISPR-modified stem cell therapy (CTX001) in 26 people with transfusion-dependent β-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, edited to produce fetal hemoglobin, aiming to reduce or elimi…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 17, 2026 02:24 UTC