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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Detalles Bibliográficos
Autores principales: Shapiro, Elsa G., Whitley, Chester B., Eisengart, Julie B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
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

Internet

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

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