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. 2009 Jan;154(1):135-9.
doi: 10.1016/j.jpeds.2008.07.004.

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

Robert F Wynn  1 , Jean Mercer, Joanne Page, Trevor F Carr, Simon Jones, J Edmond Wraith
Affiliations

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

Robert F Wynn et al. J Pediatr. 2009 Jan.

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.

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