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

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 he...

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Veröffentlicht in:	The Journal of pediatrics 2009-04, Vol.154 (4), p.609-611
Hauptverfasser:	Wynn, Robert F., MD, Wraith, J. Ed, FRCPCH, Mercer, Jean, O'Meara, Anne, FRCPI, Tylee, Karen, BSc, Thornley, Margaret, Church, Heather J., PhD, Bigger, Brian W., PhD
Format:	Artikel
Sprache:	eng
Schlagworte:	Biological and medical sciences Biomarkers - urine Chondroitin Sulfates - urine Dermatan Sulfate - urine Enzymes - blood Enzymes - therapeutic use General aspects Hematopoietic Stem Cell Transplantation Humans Medical sciences Mucopolysaccharidosis I - therapy Mucopolysaccharidosis II - therapy Mucopolysaccharidosis VI - therapy Pediatrics
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Zusammenfassung:	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.
ISSN:	0022-3476 1097-6833
DOI:	10.1016/j.jpeds.2008.11.005