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Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres.
Ghosh, Arunabha; Miller, Weston; Orchard, Paul J; Jones, Simon A; Mercer, Jean; Church, Heather J; Tylee, Karen; Lund, Troy; Bigger, Brian W; Tolar, Jakub; Wynn, Robert F.
Afiliação
  • Ghosh A; Manchester Centre For Genomic Medicine, St Mary's Hospital, Oxford Road, Manchester M13 9WL, UK; MPS Stem Cell and Neuropathies, Stopford Building, University of Manchester, Oxford Road, Manchester M13 9PT, UK. Electronic address: arunabha.ghosh@cmft.nhs.uk.
  • Miller W; Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, 2450 Riverside Avenue, Minneapolis, MN 55454, USA. Electronic address: mill4991@umn.edu.
  • Orchard PJ; Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, 2450 Riverside Avenue, Minneapolis, MN 55454, USA. Electronic address: orcha001@umn.edu.
  • Jones SA; Manchester Centre For Genomic Medicine, St Mary's Hospital, Oxford Road, Manchester M13 9WL, UK. Electronic address: simon.jones@cmft.nhs.uk.
  • Mercer J; Manchester Centre For Genomic Medicine, St Mary's Hospital, Oxford Road, Manchester M13 9WL, UK. Electronic address: jean.mercer@cmft.nhs.uk.
  • Church HJ; Manchester Centre For Genomic Medicine, St Mary's Hospital, Oxford Road, Manchester M13 9WL, UK. Electronic address: heather.church@cmft.nhs.uk.
  • Tylee K; Manchester Centre For Genomic Medicine, St Mary's Hospital, Oxford Road, Manchester M13 9WL, UK. Electronic address: karen.tylee@cmft.nhs.uk.
  • Lund T; Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, 2450 Riverside Avenue, Minneapolis, MN 55454, USA. Electronic address: lundx072@umn.edu.
  • Bigger BW; MPS Stem Cell and Neuropathies, Stopford Building, University of Manchester, Oxford Road, Manchester M13 9PT, UK. Electronic address: brian.bigger@manchester.ac.uk.
  • Tolar J; Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, 2450 Riverside Avenue, Minneapolis, MN 55454, USA. Electronic address: tolar003@umn.edu.
  • Wynn RF; Bone Marrow Transplantation Unit, Royal Manchester Children's Hospital, Oxford Road, Manchester M13 9WL, UK. Electronic address: robert.wynn@cmft.nhs.uk.
Mol Genet Metab ; 117(3): 373-7, 2016 Mar.
Article em En | MEDLINE | ID: mdl-26832957
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Transplante de Células-Tronco Hematopoéticas / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Diagnostic_studies / Etiology_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2016 Tipo de documento: Article
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Transplante de Células-Tronco Hematopoéticas / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Diagnostic_studies / Etiology_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2016 Tipo de documento: Article