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Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study.
van Gelder, C M; Poelman, E; Plug, I; Hoogeveen-Westerveld, M; van der Beek, N A M E; Reuser, A J J; van der Ploeg, A T.
Afiliación
  • van Gelder CM; Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands.
  • Poelman E; Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands.
  • Plug I; Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands.
  • Hoogeveen-Westerveld M; Department of Clinical Genetics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands.
  • van der Beek NAME; Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands.
  • Reuser AJJ; Department of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands.
  • van der Ploeg AT; Department of Clinical Genetics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands.
J Inherit Metab Dis ; 39(3): 383-390, 2016 05.
Article en En | MEDLINE | ID: mdl-26768149

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Enfermedad del Almacenamiento de Glucógeno Tipo II / Alfa-Glucosidasas Límite: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: J Inherit Metab Dis Año: 2016 Tipo del documento: Article País de afiliación: Países Bajos

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Enfermedad del Almacenamiento de Glucógeno Tipo II / Alfa-Glucosidasas Límite: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: J Inherit Metab Dis Año: 2016 Tipo del documento: Article País de afiliación: Países Bajos