A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
Status:
Completed
Trial end date:
2011-03-01
Target enrollment:
Participant gender:
Summary
This multicenter, open-label study is designed to assess safety, dose-response using
pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in
subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).