MPS-IH (HURLER SYNDROME)

Clinical trials for MPS-IH (HURLER SYNDROME) explained in plain language.

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Gene therapy trial offers new hope for children with devastating genetic disease

Disease control Ongoing

This study is testing whether a new gene therapy called OTL-203 works better than the current standard treatment (stem cell transplant) for children with Hurler syndrome, a severe genetic disorder. About 40 children will be randomly assigned to receive either the gene therapy or …

Matched conditions: MPS-IH (HURLER SYNDROME)

Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control

Last updated Apr 04, 2026 09:36 UTC

MPS-IH (HURLER SYNDROME)

Get alerted when new MPS-IH (HURLER SYNDROME) trials appear

Gene therapy trial offers new hope for children with devastating genetic disease