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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Pubblicato in:Pediatr Res
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group US 2019
Soggetti:
Clinical Research Article
Accesso online: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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