title: Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome.
abstract: We report the long-term follow-up of successful treatment of mucopolysaccharidosis type I H (MPS IH, Hurler syndrome) with combined enzyme replacement therapy and haematopoietic progenitor stem cell transplant.
authors.affiliation: Department of Genetic Metabolic Medicine, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.
authors.forename: Sunita
authors.initials: S
authors.lastname: Bijarnia
authors.forename: Peter
authors.initials: P
authors.lastname: Shaw
authors.forename: Anne
authors.initials: A
authors.lastname: Vimpani
authors.forename: Robert
authors.initials: R
authors.lastname: Smith
authors.forename: Verity
authors.initials: V
authors.lastname: Pacey
authors.forename: Helen
authors.initials: H
authors.lastname: O'Grady
authors.forename: John
authors.initials: J
authors.lastname: Christodoulou
authors.forename: David
authors.initials: D
authors.lastname: Sillence
mesh_terms: D002675:Child, Preschool; D003131:Combined Modality Therapy; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007068:Iduronidase / Q000172:deficiency / Q000627:therapeutic use*; D007223:Infant; D008059:Mucopolysaccharidosis I / Q000175:diagnosis / Q000188:drug therapy* / Q000201:enzymology / Q000601:surgery*; D016896:Treatment Outcome
chemical_list: D007068:Iduronidase