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Hurdles in treating Hurler disease: potential routes to achieve a "real" cure.
van den Broek, Brigitte T A; van Doorn, Jaap; Hegeman, Charlotte V; Nierkens, Stefan; Lindemans, Caroline A; Verhoeven-Duif, Nanda; Boelens, Jaap Jan; van Hasselt, Peter M.
Afiliação
  • van den Broek BTA; Sylvia Toth Center for Multidisciplinary Follow-up after Hematopoietic Cell Transplantation.
  • van Doorn J; Pediatric Bone and Marrow Transplantation Program, and.
  • Hegeman CV; Department of Pediatric Metabolic Diseases, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
  • Nierkens S; Section of Metabolic Diagnostics, Department of Genetics, and.
  • Lindemans CA; Sylvia Toth Center for Multidisciplinary Follow-up after Hematopoietic Cell Transplantation.
  • Verhoeven-Duif N; Center for Translational Immunology, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
  • Boelens JJ; Princess Maxima Center for Pediatric Oncology, Utrecht, The Netherlands; and.
  • van Hasselt PM; Sylvia Toth Center for Multidisciplinary Follow-up after Hematopoietic Cell Transplantation.
Blood Adv ; 4(12): 2837-2849, 2020 06 23.
Article em En | MEDLINE | ID: mdl-32574368

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Mucopolissacaridoses / Transplante de Células-Tronco Hematopoéticas Tipo de estudo: Guideline / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Mucopolissacaridoses / Transplante de Células-Tronco Hematopoéticas Tipo de estudo: Guideline / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article