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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Detalhes bibliográficos
Publicado no:Pediatr Res
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group US 2019
Assuntos:
Clinical Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6960090/
https://ncbi.nlm.nih.gov/pubmed/31434105
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41390-019-0541-2
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