MPS-IH (Hurler Syndrome) research hub

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

This study looks at a new gene therapy, OTL-203, for children with Hurler Syndrome (MPS-IH). It compares this new treatment to the standard stem cell transplant to see which works better and is safer for these young patients.