Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.

Wynn, Robert F; Mercer, Jean; Page, Joanne; Carr, Trevor F; Jones, Simon; Wraith, J Edmond

Wynn, Robert F; Mercer, Jean; Page, Joanne; Carr, Trevor F; Jones, Simon; Wraith, J Edmond.

Affiliation

Wynn RF; Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK. robert.wynn@cmmc.nhs.uk

J Pediatr ; 154(1): 135-9, 2009 Jan.

Article in En | MEDLINE | ID: mdl-19187736

Subject(s)

Hematopoietic Stem Cell Transplantation; Iduronidase/administration & dosage; Mucopolysaccharidosis I/surgery; Neoadjuvant Therapy; Female; Humans; Infant; Male; Mucopolysaccharidosis I/mortality; Survival Analysis; Transplantation Conditioning; Treatment Outcome

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Mucopolysaccharidosis I / Hematopoietic Stem Cell Transplantation / Neoadjuvant Therapy / Iduronidase Limits: Female / Humans / Infant / Male Language: En Journal: J Pediatr Year: 2009 Type: Article Affiliation country: United kingdom

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