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Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.
Potter, Jane E; Petts, Gemma; Ghosh, Arunabha; White, Fiona J; Kinsella, Jane L; Hughes, Stephen; Roberts, Jane; Hodgkinson, Adam; Brammeier, Kathryn; Church, Heather; Merrigan, Christine; Hughes, Joanne; Evans, Pamela; Campbell, Helen; Bonney, Denise; Newman, William G; Bigger, Brian W; Broomfield, Alexander; Jones, Simon A; Wynn, Robert F.
Afiliação
  • Potter JE; Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital, Oxford Road, Manchester, UK. janeelizabeth.potter@mft.nhs.uk.
  • Petts G; Department of Paediatric Histopathology, Royal Manchester Children's Hospital, Oxford Road, Manchester, UK.
  • Ghosh A; Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
  • White FJ; Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
  • Kinsella JL; Department of Therapy and Dietetics, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
  • Hughes S; Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital, Oxford Road, Manchester, UK.
  • Roberts J; Paediatric Allergy and Immunology Department, Royal Manchester Children's Hospital, Oxford Road, Manchester, UK.
  • Hodgkinson A; Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
  • Brammeier K; Department of Paediatric Histopathology, Royal Manchester Children's Hospital, Oxford Road, Manchester, UK.
  • Church H; Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
  • Merrigan C; Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
  • Hughes J; National Centre for Inherited Metabolic Disorders, Children's Health Ireland at Temple Street, Dublin, Ireland.
  • Evans P; National Centre for Inherited Metabolic Disorders, Children's Health Ireland at Temple Street, Dublin, Ireland.
  • Campbell H; Department of Haematology and Oncology, Children's Health Ireland at Crumlin, Dublin, Ireland.
  • Bonney D; Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital, Oxford Road, Manchester, UK.
  • Newman WG; Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital, Oxford Road, Manchester, UK.
  • Bigger BW; Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
  • Broomfield A; Evolution and Genomic Science, School of Biological Sciences, University of Manchester, Manchester, UK.
  • Jones SA; Stem Cell and Neurotherapies Laboratory, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, UK.
  • Wynn RF; Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Oxford Road, Manchester, UK.
Orphanet J Rare Dis ; 16(1): 235, 2021 05 21.
Article em En | MEDLINE | ID: mdl-34020687
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Wolman / Transplante de Células-Tronco Hematopoéticas / Terapia de Reposição de Enzimas Aspecto: Patient_preference Limite: Humans / Infant Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Reino Unido
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Wolman / Transplante de Células-Tronco Hematopoéticas / Terapia de Reposição de Enzimas Aspecto: Patient_preference Limite: Humans / Infant Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Reino Unido