Long-term galsulfase enzyme replacement therapy in Taiwanese mucopolysaccharidosis VI patients: A case series

Long-term galsulfase enzyme replacement therapy in Taiwanese mucopolysaccharidosis VI patients: A case series

Background: Information regarding the long-term outcome of enzyme replacement therapy (ERT) with recombinant human N-acetylgalactosamine 4-sulfatase (rhASB, galsulfase, Naglazyme®, BioMarin Pharmaceutical Inc.) for Taiwanese patients with mucopolysaccharidosis (MPS) VI is limited. Methods: Nine Taiw...

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Main Authors:	Hsiang-Yu Lin, Chih-Kuang Chuang, Chung-Hsing Wang, Yin-Hsiu Chien, Yu-Mei Wang, Fuu-Jen Tsai, Yen-Yin Chou, Shio Jean Lin, Hui-Ping Pan, Dau-Ming Niu, Wuh-Liang Hwu, Yu-Yuan Ke, Shuan-Pei Lin
Format:	Article
Language:	English
Published:	Elsevier 2016-06-01
Series:	Molecular Genetics and Metabolism Reports
Subjects:	Cardiac hypertrophy Diastolic dysfunction Enzyme replacement therapy Glycosaminoglycans Mucopolysaccharidosis VI Pulmonary function
Online Access:	http://www.sciencedirect.com/science/article/pii/S2214426916300283

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