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Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes.
Hughes, Derralynn A; Bichet, Daniel G; Giugliani, Roberto; Hopkin, Robert J; Krusinska, Eva; Nicholls, Kathleen; Olivotto, Iacopo; Feldt-Rasmussen, Ulla; Sakai, Norio; Skuban, Nina; Sunder-Plassmann, Gere; Torra, Roser; Wilcox, William R.
Afiliação
  • Hughes DA; Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust and University College London, London, UK derralynnhughes@nhs.net.
  • Bichet DG; Hôpital du Sacré Coeur, University of Montréal, Montréal, Quebec, Canada.
  • Giugliani R; Medical Genetics Service, HCPA, Department of Genetics, UFRGS, DASA and INAGEMP, Porto Alegre, Brazil.
  • Hopkin RJ; Division of Human Genetics, College of Medicine, Department of Pediatrics, Cincinnati Children's Hospital and University of Cincinnati, Cincinnati, Ohio, USA.
  • Krusinska E; Clinical Development, Amicus Therapeutics Inc, Philadelphia, Pennsylvania, USA.
  • Nicholls K; Department of Nephrology, Royal Melbourne Hospital, University of Melbourne, Parkville, Victoria, Australia.
  • Olivotto I; Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy.
  • Feldt-Rasmussen U; Department of Medical Endocrinology and Metabolism, Rigshospitalet, National University Hospital, Copenhagen University, Copenhagen, Denmark.
  • Sakai N; Department of Pediatrics, Osaka University Graduate School of Medicine, Suita, Japan.
  • Skuban N; Clinical Development, Amicus Therapeutics Inc, Philadelphia, Pennsylvania, USA.
  • Sunder-Plassmann G; Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna, Vienna, Austria.
  • Torra R; Inherited Renal Disorders, Nephrology Department, Fundació Puigvert, IIB Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Wilcox WR; Department of Human Genetics, Emory University, Atlanta, Georgia, USA.
J Med Genet ; 60(7): 722-731, 2023 07.
Article em En | MEDLINE | ID: mdl-36543533
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Fabry Tipo de estudo: Prognostic_studies / Risk_factors_studies Limite: Female / 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 Fabry Tipo de estudo: Prognostic_studies / Risk_factors_studies Limite: Female / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article