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Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)
BACKGROUND: Mucopolysaccharidosis II, also known as Hunter syndrome, is a rare, X‐linked disease caused by a deficiency of the lysosomal enzyme iduronate‐2‐sulfatase, which catalyses a step in the catabolism of glycosaminoglycans. The glycosaminoglycans accumulate within tissues affecting multiple o...
保存先:
| 出版年: | Cochrane Database Syst Rev |
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| 主要な著者: | , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
John Wiley & Sons, Ltd
2016
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| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7173756/ https://ncbi.nlm.nih.gov/pubmed/26845288 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD008185.pub4 |
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