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Hunter syndrome follow-up after 1 year of enzyme-replacement therapy
Mucopolysaccharidosis II (Hunter syndrome) is a rare x-linked disorder caused by a deficiency in the lysosomal enzyme iduronate-2-sulphatase, leading to an accumulation of the glycosaminoglycans (GAGs) dermatansulphate and heparan sulphate. The consequence of GAGs accumulation is progressive, multio...
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| Hoofdauteurs: | , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
BMJ Publishing Group
2013
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3603921/ https://ncbi.nlm.nih.gov/pubmed/23307460 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/bcr-2012-007644 |
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