MUCOPOLYSACCHARIDOSIS IH

Clinical trials for MUCOPOLYSACCHARIDOSIS IH explained in plain language.

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One-Time treatment aims to halt devastating childhood disease

Disease control Ongoing

This study is testing a one-time gene therapy for children with Hurler syndrome, a severe and life-limiting genetic disorder. Doctors collect a child's own blood stem cells, add a corrected gene in the lab, and then return the cells to the child. The main goals are to see if this…

Matched conditions: MUCOPOLYSACCHARIDOSIS IH

Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control

Last updated Mar 30, 2026 14:35 UTC

MUCOPOLYSACCHARIDOSIS IH

Get alerted when new MUCOPOLYSACCHARIDOSIS IH trials appear

One-Time treatment aims to halt devastating childhood disease