A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell trans

Medicine & Life Sciences

1. Mucopolysaccharidosis I 100%
2. Hematopoietic Stem Cells 34%
3. Standard of Care 33%
4. Randomized Controlled Trials 23%
5. Safety 20%