Presentation and Treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome)

INTRODUCTION: Mucopolysaccharidosis Type II (MPS II; Hunter syndrome) is an X- linked lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS). IDS deficiency leads to primary accumulation of dermatan sulfate (DS) and heparan sulfate (HS). MPS II is both multi-systemic and pr...

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Pubblicato in:Expert Opin Orphan Drugs
Autori principali: Stapleton, Molly, Kubaski, Francyne, Mason, Robert W., Yabe, Hiromasa, Suzuki, Yasuyuki, Orii, Kenji E., Orii, Tadao, Tomatsu, Shunji
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2017
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5693349/
https://ncbi.nlm.nih.gov/pubmed/29158997
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/21678707.2017.1296761
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