Mucopolysaccharidosis I
Not recruitingStudy on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation
Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation
- Disease:Mucopolysaccharidosis I
- Substances:BusulfanFludarabine+4
Italy- Participants:0–17 years
- Substances:Busulfan
- Sponsor:Orchard Therapeutics (Europe) Limited
















