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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書目詳細資料
發表在:JIMD Rep
Main Authors: Haller, Christine, Song, Wenjie, Cimms, Tricia, Chen, Chao‐Yin, Whitley, Chester B., Wang, Raymond Y., Bauer, Mislen, Harmatz, Paul
格式: Artigo
語言:Inglês
出版: John Wiley & Sons, Inc. 2019
主題:
Research Reports
在線閱讀: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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