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Clinical Trial
. 2009 Apr;154(4):609-11.
doi: 10.1016/j.jpeds.2008.11.005.

Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy

Robert F Wynn  1 , J Ed Wraith, Jean Mercer, Anne O'Meara, Karen Tylee, Margaret Thornley, Heather J Church, Brian W Bigger
Affiliations
Clinical Trial

Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy

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

Abstract

We compared substrate reduction in patients with lysosomal storage disorder treated with hematopoietic stem cell transplant and found that it was significantly reduced compared with patients treated with pharmacological enzyme replacement therapy. These data might support the wider application of hematopoietic stem cell transplant in the treatment of lysosomal storage disorders.

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