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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

 
Wynn RF, Mercer J, Page J, Carr TF, Jones S, Wraith JE 
Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. [Journal Article, Research Support, Non-U.S. Gov't]
J Pediatr 2009 Jan; 154(1):135-9.


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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