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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Detalles Bibliográficos
Autores principales: Haller, Christine, Song, Wenjie, Cimms, Tricia, Chen, Chao‐Yin, Whitley, Chester B., Wang, Raymond Y., Bauer, Mislen, Harmatz, Paul
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2019
Materias:
Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6718107/
https://www.ncbi.nlm.nih.gov/pubmed/31497482
http://dx.doi.org/10.1002/jmd2.12043

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