Not recruiting
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
Registered drugNo placebo
Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation
Registered drugNo placebo
- Disease:Mucopolysaccharidosis I
- Substances:BusulfanFludarabine+4
Countries:
Italy
Italy- Participants:0–17 years
- Substances:Busulfan

