Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. The Journal of pediatrics [J Pediatr] Journal article

Title	Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.
Author(s)	Wynn RF, Mercer J, Page J, Carr TF, Jones S, Wraith JE
Institution	Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK. robert.wynn@cmmc.nhs.uk
Source	J Pediatr 2009 Jan; 154(1):135-9.
MeSH	Female Hematopoietic Stem Cell Transplantation Humans Iduronidase Infant Male Mucopolysaccharidosis I Neoadjuvant Therapy Survival Analysis Transplantation Conditioning Treatment Outcome
Abstract	We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning.
Language	eng
Pub Type(s)	Journal Article Research Support, Non-U.S. Gov't
PubMed ID	19187736

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