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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Bibliographic Details

Published in:	Molecular Genetics and Metabolism Reports
Main Authors:	Mei-Yan Chan, Andrew Jack Nelson, Lock-Hock Ngu
Format:	Article
Language:	English
Published:	Elsevier 2023-09-01
Subjects:	Mucopolysaccharidosis Asian Enzyme replacement therapy Idursulfase beta
Online Access:	http://www.sciencedirect.com/science/article/pii/S221442692300037X