Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome.
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.
Bijarnia
Sunita
S
Department of Genetic Metabolic Medicine, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.
Shaw
Peter
P
Vimpani
Anne
A
Smith
Robert
R
Pacey
Verity
V
O'Grady
Helen
H
Christodoulou
John
J
Sillence
David
D
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
D007068:Iduronidase