Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population

Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population

Abstract Mucopolysaccharidosis (MPS) VII is an ultra‐rare, progressively debilitating, life‐threatening lysosomal disease caused by deficiency of the enzyme, β‐glucuronidase. Vestronidase alfa is an approved enzyme replacement therapy for MPS VII. UX003‐CL301 was a phase 3, randomized, placebo‐contr...

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Bibliographic Details
Main Authors: Christine Haller, Wenjie Song, Tricia Cimms, Chao‐Yin Chen, Chester B. Whitley, Raymond Y. Wang, Mislen Bauer, Paul Harmatz
Format: Article
Language:English
Published: Wiley 2019-09-01
Series:JIMD Reports
Subjects:
enzyme replacement therapy
heat map
MPS VII
mucopolysaccharidosis
Sly syndrome
vestronidase alfa
Online Access:https://doi.org/10.1002/jmd2.12043

Internet

https://doi.org/10.1002/jmd2.12043

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