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Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison.
Eisengart, Julie B; Rudser, Kyle D; Xue, Yong; Orchard, Paul; Miller, Weston; Lund, Troy; Van der Ploeg, Ans; Mercer, Jean; Jones, Simon; Mengel, Karl Eugen; Gökce, Seyfullah; Guffon, Nathalie; Giugliani, Roberto; de Souza, Carolina F M; Shapiro, Elsa G; Whitley, Chester B.
Afiliación
  • Eisengart JB; Division of Clinical Behavioral Neuroscience, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA. eisen139@umn.edu.
  • Rudser KD; Division of Biostatistics, University of Minnesota, Minneapolis, Minnesota, USA.
  • Xue Y; Sanofi Genzyme Corporation, Naarden, the Netherlands.
  • Orchard P; Division of Hematology/Oncology/Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
  • Miller W; Division of Hematology/Oncology/Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
  • Lund T; Division of Hematology/Oncology/Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
  • Van der Ploeg A; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Hospital, Rotterdam, The Netherlands.
  • Mercer J; Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, Manchester, UK.
  • Jones S; Departments of Haematology and BMT, Royal Manchester Children's Hospital, Manchester, UK.
  • Mengel KE; Department of Pediatric and Adolescent Medicine, Villa Metabolica, University Medical Center-Mainz, Mainz, Germany.
  • Gökce S; Department of Pediatric and Adolescent Medicine, Villa Metabolica, University Medical Center-Mainz, Mainz, Germany.
  • Guffon N; Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Femme Mère Enfant, Bron Cedex, France.
  • Giugliani R; Department of Genetics, Federal University of Rio Grande do Sul and Medical Genetics Service, Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil.
  • de Souza CFM; Department of Genetics, Federal University of Rio Grande do Sul and Medical Genetics Service, Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil.
  • Shapiro EG; Division of Clinical Behavioral Neuroscience, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
  • Whitley CB; Shapiro Neuropsychology Consulting, Portland, Oregon, USA.
Genet Med ; 20(11): 1423-1429, 2018 11.
Article en En | MEDLINE | ID: mdl-29517765
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Tamizaje Neonatal / Mucopolisacaridosis I / Trasplante de Células Madre Hematopoyéticas / Terapia de Reemplazo Enzimático Tipo de estudio: Clinical_trials / Diagnostic_studies / Guideline / Prognostic_studies Aspecto: Ethics Límite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Tamizaje Neonatal / Mucopolisacaridosis I / Trasplante de Células Madre Hematopoyéticas / Terapia de Reemplazo Enzimático Tipo de estudio: Clinical_trials / Diagnostic_studies / Guideline / Prognostic_studies Aspecto: Ethics Límite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos