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Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study.
Ditters, Imke A M; van der Beek, Nadine A M E; Brusse, Esther; van der Ploeg, Ans T; van den Hout, Johanna M P; Huidekoper, Hidde H.
Afiliação
  • Ditters IAM; Department of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.
  • van der Beek NAME; Department of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands.
  • Brusse E; Department of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands.
  • van der Ploeg AT; Department of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.
  • van den Hout JMP; Department of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.
  • Huidekoper HH; Department of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands. h.huidekoper@erasmusmc.nl.
Orphanet J Rare Dis ; 18(1): 108, 2023 05 08.
Article em En | MEDLINE | ID: mdl-37158969
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Adult / Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Adult / Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article