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Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series

Mucopolysaccharidosis (MPS) type II (Hunter syndrome) is a rare X-linked, recessive, lysosomal storage disorder caused by the deficit of the enzyme iduronate 2-sulfatase (IDS), resulting in accumulation of glycosaminoglycans (GAGs) impairing cellular function in multiple organ systems. Idursulfase (...

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Detalles Bibliográficos
Autores principales:	Chan, Mei-Yan, Nelson, Andrew Jack, Ngu, Lock-Hock
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2023
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10475840/ https://www.ncbi.nlm.nih.gov/pubmed/37670899 http://dx.doi.org/10.1016/j.ymgmr.2023.100991

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