Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
Status:
Recruiting
Trial end date:
2029-10-31
Target enrollment:
Participant gender:
Summary
A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and
explore efficacy of the study drug in development for the treatment of MPS IIIA patients.