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Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
PURPOSE: Abnormalities in cerebrospinal fluid (CSF) have been reported in Hurler syndrome, a fatal neurodegenerative lysosomal disorder. While no biomarker has predicted neurocognitive response to treatment, one of these abnormalities, glycosaminoglycan nonreducing ends (NREs), holds promise to moni...
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| Publicado no: | Genet Med |
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| Main Authors: | , , , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group US
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6831510/ https://ncbi.nlm.nih.gov/pubmed/31019279 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41436-019-0522-1 |
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