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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Bibliografiske detaljer
Udgivet i:Mol Genet Metab Rep
Main Authors: Lin, Hsiang-Yu, Chuang, Chih-Kuang, Wang, Chung-Hsing, Chien, Yin-Hsiu, Wang, Yu-Mei, Tsai, Fuu-Jen, Chou, Yen-Yin, Lin, Shio Jean, Pan, Hui-Ping, Niu, Dau-Ming, Hwu, Wuh-Liang, Ke, Yu-Yuan, Lin, Shuan-Pei
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2016
Fag:
Research Paper
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4834679/
https://ncbi.nlm.nih.gov/pubmed/27134829
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2016.04.003
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