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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Bibliografiset tiedot
Julkaisussa:Pediatr Res
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Nature Publishing Group US 2019
Aiheet:
Clinical Research Article
Linkit: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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