Mucopolysaccharidosis II Clinical Trial
Official title:
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below, - to collect the safety information of JR-141 - to evaluate the plasma pharmacokinetics of JR-141 - to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms
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