HURLER SYNDROME
Clinical trials for HURLER SYNDROME explained in plain language.
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Gene therapy trial aims to treat rare brain disease MPS i
Disease control TerminatedThis study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disorder 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 tolerabl…
Matched conditions: HURLER SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 17, 2026 12:57 UTC
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New stem cell approach aims to tame rare genetic diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get donor cells to grow in the patient's body while keeping transplant-related deaths low. Participants receive chemotherapy drugs (busulfan and f…
Matched conditions: HURLER SYNDROME
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 12, 2026 13:39 UTC