A Study of JR-171 in Patients With Mucopolysaccharidosis I
Status:
Recruiting
Trial end date:
2022-05-31
Target enrollment:
Participant gender:
Summary
Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to
evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of
mucopolysaccharidosis type I (MPS I).