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

@article{Tolar2008CombinationOE,
  title={Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome},
  author={Jakub Tolar and Satkiran S. Grewal and Kendra J Bjoraker and Chester B. Whitley and Elena G. Shapiro and Lawrence Charnas and Paul J Orchard},
  journal={Bone Marrow Transplantation},
  year={2008},
  volume={41},
  pages={531-535}
}
Hurler syndrome (mucopolysaccharidosis type I, MPS IH) is characterized by a deficiency of α-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 MPS IH. Seven children with MPS IH at a median age of 1.5 years at the time of myeloablative HSCT were eligible. All patients had null mutations in IDUA gene… CONTINUE READING
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