Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

Research output: Contribution to journalArticle

  • External authors:
  • Robert F. Wynn
  • J. Ed Wraith
  • Jean Mercer
  • Anne O'Meara
  • Karen Tylee
  • Margaret Thornley
  • Heather J. Church


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. © 2009 Mosby, Inc. All rights reserved.

Bibliographical metadata

Original languageEnglish
Pages (from-to)609-611
Number of pages2
JournalJournal Of Pediatrics
Issue number4
Publication statusPublished - Apr 2009

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