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Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy |
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| Authors: | Robert F. Wynn J. Ed Wraith Jean Mercer Karen Tylee Margaret Thornley Brian W. Bigger |
| Affiliation: | a Departments of Haematology and BMT, Royal Manchester Children's Hospital, Manchester, United Kingdom b Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, Manchester, United Kingdom c Department of Paediatric Oncology, Our Lady's Children's Hospital, Dublin, Ireland d MPS Stem Cell Research Laboratory, University of Manchester, Manchester, United Kingdom |
| Abstract: | |
| Keywords: | DS/CS, Dermatan sulphate to chondroitin sulphate ERT, Enzyme replacement therapy HSCT, Hematopoietic stem cell transplant LSD, Lysosomal storage disorder MPSIH, Mucopolysaccharidosis type IH |
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