HURLER SYNDROME
Clinical trials for HURLER SYNDROME explained in plain language.
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Gene therapy trial for rare childhood disease halted Mid-Study
Disease control TerminatedThis study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that damages the brain and body. The therapy delivers a working copy of the missing IDUA gene directly into the fluid around the brain. The trial aims to check if the treatment is safe a…
Matched conditions: HURLER SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:01 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 the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…
Matched conditions: HURLER SYNDROME
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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New algorithm aims to catch ovarian damage early in young cancer survivors
Knowledge-focused TerminatedThis study was designed to see if a new monitoring algorithm could detect early signs of ovarian damage in women and girls who had cancer treatment. Researchers planned to compare a group using the new algorithm with a group that received standard care. However, the study was wit…
Matched conditions: HURLER SYNDROME
Sponsor: University of Colorado, Denver • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:42 UTC