Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome

OBJECTIVE: To investigate whether intravenous enzyme replacement therapy (ERT) benefits cognitive function in patients with mucopolysaccharidosis Type IH (Hurler syndrome, MPS IH) undergoing hematopoietic cell transplantation (HCT). STUDY DESIGN: Data were obtained for nine children treated with HCT...

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Autors principals: Eisengart, Julie B., Rudser, Kyle D., Tolar, Jakub, Orchard, Paul J., Kivisto, Teresa, Ziegler, Richard S., Whitley, Chester, Shapiro, Elsa
Format: Artigo
Idioma:Inglês
Publicat: 2012
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3524404/
https://ncbi.nlm.nih.gov/pubmed/22974573
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jpeds.2012.07.052
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