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Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.
Ditters, Imke Anne Maartje; Huidekoper, Hidde Harmen; Kruijshaar, Michelle Elisabeth; Rizopoulos, Dimitris; Hahn, Andreas; Mongini, Tiziana Enrica; Labarthe, François; Tardieu, Marine; Chabrol, Brigitte; Brassier, Anais; Parini, Rossella; Parenti, Giancarlo; van der Beek, Nadine Anna Maria Elisabeth; van der Ploeg, Ans Tjitske; van den Hout, Johanna Maria Pieternel.
Afiliação
  • Ditters IAM; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Huidekoper HH; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Kruijshaar ME; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Rizopoulos D; Center for Lysosomal and Metabolic Diseases, and Department of Biostatistics, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Hahn A; Department of Child Neurology, University of Giessen, Giessen, Germany.
  • Mongini TE; Neuromuscular Center, Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino, University of Turin, Turin, Italy.
  • Labarthe F; Department of Pediatrics, Center for Inborn Errors of Metabolism ToTeM, Centre Hospitalier Régional Universitaire de Tours, Tours, France.
  • Tardieu M; Department of Pediatrics, Center for Inborn Errors of Metabolism ToTeM, Centre Hospitalier Régional Universitaire de Tours, Tours, France.
  • Chabrol B; Department of Pediatrics, University Hospital of Marseille, Marseille, France.
  • Brassier A; Department of Pediatric Neurology, Hôpital Necker-Enfants Malades, Paris, France.
  • Parini R; Department of Pediatrics, Unit of Rare Metabolic Diseases, San Gerardo Hospital, Monza, Italy.
  • Parenti G; Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy; Telethon Institute of Genetics and Medicine, Naples, Italy.
  • van der Beek NAME; Department of Neurology, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • van der Ploeg AT; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • van den Hout JMP; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, Netherlands. Electronic address: j.vandenhout@erasmusmc.nl.
Lancet Child Adolesc Health ; 6(1): 28-37, 2022 01.
Article em En | MEDLINE | ID: mdl-34822769
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Caminhada / Relação Dose-Resposta a Droga / Alfa-Glucosidases / Terapia de Reposição de Enzimas País/Região como assunto: Europa Idioma: En Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Holanda
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Caminhada / Relação Dose-Resposta a Droga / Alfa-Glucosidases / Terapia de Reposição de Enzimas País/Região como assunto: Europa Idioma: En Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Holanda