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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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| 出版年: | Expert Opin Orphan Drugs |
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| 主要な著者: | , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2017
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| 主題: | |
| オンライン･アクセス: | 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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