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

Robert F. Wynn; J. Ed Wraith; Jean Mercer; Anne O'Meara; Karen Tylee; Margaret Thornley; Heather J. Church; Brian W. Bigger

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, J. Ed Wraith, Jean Mercer, Anne O'Meara, Karen Tylee, Margaret Thornley, Heather J. Church, Brian W. Bigger

Research output: Contribution to journal › Article › peer-review

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. © 2009 Mosby, Inc. All rights reserved.

Original language	English
Pages (from-to)	609-611
Number of pages	2
Journal	Journal Of Pediatrics
Volume	154
Issue number	4
DOIs	https://doi.org/10.1016/j.jpeds.2008.11.005
Publication status	Published - Apr 2009

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10.1016/j.jpeds.2008.11.005

Improved clinical management of lysosomal disorders
Wraith, J. E. (Participant), Fairbairn, L. J. (Participant), (Participant), Bigger, B. (Participant) & (Participant)
Impact: Health impacts

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

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. {\textcopyright} 2009 Mosby, Inc. All rights reserved.",

author = "Wynn, {Robert F.} and Wraith, {J. Ed} and Jean Mercer and Anne O'Meara and Karen Tylee and Margaret Thornley and Church, {Heather J.} and Bigger, {Brian W.}",

year = "2009",

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AU - Wynn, Robert F.

AU - Wraith, J. Ed

AU - Mercer, Jean

AU - O'Meara, Anne

AU - Tylee, Karen

AU - Thornley, Margaret

AU - Church, Heather J.

AU - Bigger, Brian W.

PY - 2009/4

Y1 - 2009/4

N2 - 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.

AB - 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.

U2 - 10.1016/j.jpeds.2008.11.005

DO - 10.1016/j.jpeds.2008.11.005

M3 - Article

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SN - 0022-3476

VL - 154

SP - 609

EP - 611

JO - Journal Of Pediatrics

JF - Journal Of Pediatrics

IS - 4

ER -

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

Abstract

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Impacts

Improved clinical management of lysosomal disorders

Cite this