The Journal of pediatrics, 2009-04, Vol.154 (4), p.609-611
2009
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Details
- Autor(en) / Beteiligte
- Titel
- Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy
- Ist Teil von
- The Journal of pediatrics, 2009-04, Vol.154 (4), p.609-611
- Ort / Verlag
- Maryland Heights, MO: Mosby, Inc
- Erscheinungsjahr
- 2009
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- 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.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0022-3476eISSN: 1097-6833DOI: 10.1016/j.jpeds.2008.11.005Titel-ID: cdi_proquest_miscellaneous_67080994
- Format
- –
- 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