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Clinical trial

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

BioMarin PharmaceuticalTerminatedStarted Oct 1, 2008353 enrolledUnited States, Argentina, Brazil, Canada, France, Germany, Italy, Netherlands, Taiwan, United KingdomNCT00787995

Abstract

This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.

Topics & conditions

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LSD

Keywords

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

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