HURLER SYNDROME
Clinical trials for HURLER SYNDROME explained in plain language.
Never miss a new study
Get alerted when new HURLER SYNDROME trials appear
Sign up with your email to follow new studies for HURLER SYNDROME, 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
-
Gene therapy trial aims to treat rare brain disease
Disease control TerminatedThis study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disease that affects the brain and body. The therapy delivers a working copy of the missing IDUA gene directly into the fluid around the brain. The main goal is to see if it is safe and tolerable…
Matched conditions: HURLER SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 26, 2026 06:21 UTC
-
New stem cell approach shows promise for rare childhood diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to see if a donor's stem cells can successfully take over the patient's bone marrow while keeping side effects low. Participants receive chemotherapy…
Matched conditions: HURLER SYNDROME
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 26, 2026 04:38 UTC