Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials

INTRODUCTION: Mucopolysaccharidosis type VII (MPS VII, Sly Syndrome) is a progressive, debilitating, ultra-rare lysosomal storage disorder caused by the deficiency of β-glucuronidase (GUS), an enzyme required for breakdown of glycosaminoglycans (GAGs). Vestronidase alfa, a recombinant human GUS, is...

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Bibliographic Details
Published in:Clin Pharmacokinet
Main Authors: Qi, Yulan, McKeever, Kathleen, Taylor, Julie, Haller, Christine, Song, Wenjie, Jones, Simon A., Shi, Jack
Format: Artigo
Language:Inglês
Published: Springer International Publishing 2018
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Original Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6451706/
https://ncbi.nlm.nih.gov/pubmed/30467742
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40262-018-0721-y
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