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

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

The Journal of pediatrics. 2009;154(4):609.

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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.

Bibliographic metadata

Content type:

Journal article

Publication type:

Original research

Publication form:

Academic journal article

Author(s):

Published date:

2009-04

Article title:

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

Language:

eng

Journal title:

The Journal of pediatrics

ISSN:

0022-3476

Publisher:

Elsevier

Place of publication:

United States

Volume:

154

Issue:

Start page:

609

Digital Object Identifier:

10.1016/j.jpeds.2008.11.005

Pubmed Identifier:

19324223

Pii Identifier:

S0022-3476(08)00962-1

Access state:

Active

Institutional metadata

University researcher(s):

Bigger, Brian

Academic department(s):

Record metadata

Manchester eScholar ID:

uk-ac-man-scw:80172

Created by:

Bigger, Brian

Created:

4th May, 2010, 16:07:08

Last modified by:

Bigger, Brian

Last modified:

20th January, 2015, 08:27:25