A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
study id #: NCT05208281
condition: Mucopolysaccharidosis Type II, Metabolic Diseases
status: Recruiting
purpose:This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
intervention: GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg
results: https://clinicaltrials.gov/ct2/show/results/NCT05208281
last updated: February 26, 2022
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