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Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.

BONE MARROW TRANSPLANTATION, no. 6.0 (2008): 531-535

Cited: 74|Views28

WOS NATURE

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mucopolysaccharidosis type IHurler syndromehematopoietic stem cell transplantationenzyme replacement therapyiduronidase

Abstract

Hurler syndrome (mucopolysaccharidosis type I, MPS IH) is characterized by a deficiency of alpha-L-iduronidase resulting in progressive multiorgan dysfunction. We sought to determine whether enzyme replacement therapy (ERT) with iduronidase in the peritransplant period affects outcome of hematopoietic stem cell transplantation (HSCT) for ...More

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J Tolar

S S Grewal

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K J Bjoraker

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C B Whitley

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E G Shapiro

L Charnas

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P J Orchard

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