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The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease - A systematic literature review by a European panel of experts.
Spada, Marco; Baron, Ralf; Elliott, Perry M; Falissard, Bruno; Hilz, Max J; Monserrat, Lorenzo; Tøndel, Camilla; Tylki-Szymanska, Anna; Wanner, Christoph; Germain, Dominique P.
Afiliação
  • Spada M; Department of Paediatrics, University of Torino, Torino, Italy. Electronic address: marco.spada@unito.it.
  • Baron R; Division of Neurological Pain Research and Therapy, Department of Neurology, Universitätsklinikum Schleswig-Holstein, Kiel, Germany.
  • Elliott PM; Barts Heart Centre, University College London, London, United Kingdom.
  • Falissard B; INSERM U1018, University of Paris-Sud, University of Paris-Descartes, Paris, France.
  • Hilz MJ; Department of Neurology, University of Erlangen-Nuremberg, Erlangen, Germany.
  • Monserrat L; Health in Code, A Coruña, Spain.
  • Tøndel C; Department of Paediatrics, Haukeland University Hospital, Department of Clinical Medicine, University of Bergen, Bergen, Norway.
  • Tylki-Szymanska A; Department of Paediatrics, Nutrition and Metabolic Diseases, Children's Memorial Health Institute, Warsaw, Poland.
  • Wanner C; Division of Nephrology, University Clinic, University of Würzburg, Würzburg, Germany.
  • Germain DP; French Referral Center for Fabry disease, Division of Medical Genetics and INSERM U1179, University of Versailles, Paris-Saclay University, Montigny, France.
Mol Genet Metab ; 126(3): 212-223, 2019 03.
Article em En | MEDLINE | ID: mdl-29785937
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Fabry / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Observational_studies / Systematic_reviews Limite: Child / Female / Humans / Male País/Região como assunto: Europa Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2019 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Fabry / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Observational_studies / Systematic_reviews Limite: Child / Female / Humans / Male País/Região como assunto: Europa Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2019 Tipo de documento: Article