Journal
JOURNAL OF PEDIATRICS
Volume 154, Issue 4, Pages 609-611Publisher
MOSBY-ELSEVIER
DOI: 10.1016/j.jpeds.2008.11.005
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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. (J Pediatr 2009;154:609-11)
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