Clinical trial

A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

BioMarin PharmaceuticalCompletedPhase 1Started Apr 1, 200920 enrolledUnited KingdomNCT00884949

Abstract

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).

Topics & conditions

Detected automatically by Psychedelic Commons from the title and abstract, not labels from the source. They may be incomplete.

LSD

Keywords

Mucopolysaccharidosis IV type AMPS IV Type AMucopolysaccharidosis IVAMPS IVAMorquio A SyndromeLysosomal Storage DisorderN-acetylgalactosamine-6-sulfataseN-acetylgalactosamine-6-sulfate sulfatasegalactose-6-sulfataseGALNSenzyme replacement therapyERTMPS IV A

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