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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...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2018
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948735/ https://www.ncbi.nlm.nih.gov/pubmed/29751845 http://dx.doi.org/10.1186/s13023-018-0817-3 |