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Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation

BACKGROUND: Mucopolysaccharidosis I (MPS IH) is a lysosomal storage disease treated with hematopoietic cell transplantation (HCT) because it stabilizes cognitive deterioration, but is insufficient to alleviate all somatic manifestations. Intravenous laronidase improves somatic burden in attenuated M...

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Detalles Bibliográficos
Autores principales:	Polgreen, Lynda E., Lund, Troy C., Braunlin, Elizabeth, Tolar, Jakub, Miller, Bradley S., Fung, Ellen, Whitley, Chester B., Eisengart, Julie B., Northrop, Elise, Rudser, Kyle, Miller, Weston P., Orchard, Paul J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group US 2019
Materias:	Clinical Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6960090/ https://www.ncbi.nlm.nih.gov/pubmed/31434105 http://dx.doi.org/10.1038/s41390-019-0541-2

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