HURLER-SCHEIE SYNDROME

Clinical trials for HURLER-SCHEIE SYNDROME explained in plain language.

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Brain-Targeting gene therapy tested for devastating rare disease

Disease control Terminated

This 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-SCHEIE SYNDROME

Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control

Last updated Mar 30, 2026 14:28 UTC

HURLER-SCHEIE SYNDROME

Get alerted when new HURLER-SCHEIE SYNDROME trials appear

Brain-Targeting gene therapy tested for devastating rare disease