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Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
BACKGROUND: Hurler syndrome (MPS IH), the severe, neurodegenerative form of type one mucopolysaccharidosis, is associated with rapid neurocognitive decline during toddlerhood and multi-system dysfunction. It is now standardly treated with hematopoietic cell transplantation (HCT), which halts accumul...
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| Publicado no: | Orphanet J Rare Dis |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5948735/ https://ncbi.nlm.nih.gov/pubmed/29751845 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0817-3 |
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