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

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‐controlled, bl...

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Bibliografische gegevens
Gepubliceerd in:JIMD Rep
Hoofdauteurs: Haller, Christine, Song, Wenjie, Cimms, Tricia, Chen, Chao‐Yin, Whitley, Chester B., Wang, Raymond Y., Bauer, Mislen, Harmatz, Paul
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: John Wiley & Sons, Inc. 2019
Onderwerpen:
Research Reports
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6718107/
https://ncbi.nlm.nih.gov/pubmed/31497482
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12043
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