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Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future
Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare, multisystemic, progressive lysosomal storage disease caused by deficient activity of the iduronate-2-sulfatase (I2S) enzyme. Accumulation of the glycosaminoglycans dermatan sulfate and heparan sulfate results in a broad...
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| Publicado no: | Drug Des Devel Ther |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Dove Medical Press
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5574592/ https://ncbi.nlm.nih.gov/pubmed/28860717 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S139601 |
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