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Low-dose agalsidase beta treatment in male pediatric patients with Fabry disease: A 5-year randomized controlled trial.
Ramaswami, Uma; Bichet, Daniel G; Clarke, Lorne A; Dostalova, Gabriela; Fainboim, Alejandro; Fellgiebel, Andreas; Forcelini, Cassiano M; An Haack, Kristina; Hopkin, Robert J; Mauer, Michael; Najafian, Behzad; Scott, C Ronald; Shankar, Suma P; Thurberg, Beth L; Tøndel, Camilla; Tylki-Szymanska, Anna; Bénichou, Bernard; Wijburg, Frits A.
Afiliação
  • Ramaswami U; Lysosomal Disorders Unit, Institute of Immunity and Transplantation, Royal Free London NHS Foundation Trust, University College of London, London, United Kingdom. Electronic address: uma.ramaswami@nhs.net.
  • Bichet DG; Nephrology Service, Research Center, Hôpital du Sacré-Coeur de Montréal and University of Montreal, Montreal, QC, Canada.
  • Clarke LA; Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada.
  • Dostalova G; 2nd Department of Internal Medicine and Department of Cardiovascular Medicine, Charles University Prague, General University Hospital Prague, Prague, Czech Republic.
  • Fainboim A; Hospital de Niños Ricardo Gutierrez, Hospital de Dia Polivalente, Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina.
  • Fellgiebel A; Department of Psychiatry and Psychotherapy, University Medical Center Mainz, Mainz, Germany.
  • Forcelini CM; Faculty of Medicine, Universidade de Passo Fundo, and Hospital São Vicente de Paulo, Passo Fundo, RS, Brazil.
  • An Haack K; Sanofi Genzyme, Chilly-Mazarin, France.
  • Hopkin RJ; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College Medicine, Cincinnati, OH, USA.
  • Mauer M; Departments of Pediatrics and Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Najafian B; Department of Pathology, University of Washington, Seattle, WA, USA.
  • Scott CR; Division of Genetic Medicine, Department of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA.
  • Shankar SP; Departments of Human Genetics and Ophthalmology, Emory University School of Medicine, Decatur, GA, USA.
  • Thurberg BL; Sanofi Genzyme, Framingham, MA, USA.
  • Tøndel C; Departments of Pediatrics and Clinical Medicine, Haukeland University Hospital, Bergen, Norway.
  • Tylki-Szymanska A; Department of Pediatric Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland.
  • Bénichou B; Formerly Sanofi Genzyme, Saint-Germain-en-Laye Cedex, France.
  • Wijburg FA; Department of Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center "Sphinx", Academic Medical Center, University Hospital of Amsterdam, Amsterdam, the Netherlands.
Mol Genet Metab ; 127(1): 86-94, 2019 05.
Article em En | MEDLINE | ID: mdl-30987917
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Fabry / Alfa-Galactosidase / Terapia de Reposição de Enzimas / Isoenzimas Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Humans / Male Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2019 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Fabry / Alfa-Galactosidase / Terapia de Reposição de Enzimas / Isoenzimas Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Humans / Male Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2019 Tipo de documento: Article