Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses.

Simon A Jones,Deborah Marsden,Tony Koutsoukos,Jennifer Sniadecki,Karen Tylee, Sarah Phillippo,Emil Kakkis

Molecular Genetics and Metabolism（2020）

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摘要

In this retrospective study, uGAG reduction was associated with long-term clinical outcomes as assessed by a number of approaches, supporting the use of uGAG reduction as a biomarker primary endpoint.

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关键词

Mucopolysaccharidosis,Retrospective medical chart review,Urinary glycosaminoglycan,Enzyme replacement therapy

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