HURLER SYNDROME
Clinical trials for HURLER SYNDROME explained in plain language.
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Brain-Targeting gene therapy tested for devastating rare disease
Disease control TerminatedThis early-stage study is testing a one-time gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that affects the brain. The therapy aims to deliver a working copy of a missing gene directly to the central nervous system to help the brain produce a needed e…
Matched conditions: HURLER SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
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New hope for kids with devastating genetic diseases
Disease control OngoingThis study is testing a stem cell transplant procedure for children and adults with severe, inherited metabolic disorders and a rare bone disease. The goal is to see if using a specific drug combination can help the donor cells successfully take hold in the patient's body while k…
Matched conditions: HURLER SYNDROME
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Mar 13, 2026 15:05 UTC