GENETIC DISEASES, X-LINKED
Clinical trials for GENETIC DISEASES, X-LINKED explained in plain language.
Never miss a new study
Get alerted when new GENETIC DISEASES, X-LINKED trials appear
Sign up with your email to follow new studies for GENETIC DISEASES, X-LINKED, 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 and improve muscle function. Participants must be at least 2 years …
Matched conditions: GENETIC DISEASES, X-LINKED
Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
-
Massive global registry aims to unlock secrets of rett syndrome
Knowledge-focused Recruiting nowThis study creates a large registry of people with Rett syndrome, a rare genetic disorder that causes severe intellectual and physical disabilities. Researchers will collect information from doctors and caregivers to understand how the disease progresses over time. The goal is to…
Matched conditions: GENETIC DISEASES, X-LINKED
Sponsor: International Rett Syndrome Foundation • Aim: Knowledge-focused
Last updated Jul 01, 2026 23:00 UTC