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Standardising clinical outcomes measures for adult clinical trials in Fabry disease: A global Delphi consensus.
Moreno-Martinez, D; Aguiar, P; Auray-Blais, C; Beck, M; Bichet, D G; Burlina, A; Cole, D; Elliott, P; Feldt-Rasmussen, U; Feriozzi, S; Fletcher, J; Giugliani, R; Jovanovic, A; Kampmann, C; Langeveld, M; Lidove, O; Linhart, A; Mauer, M; Moon, J C; Muir, A; Nowak, A; Oliveira, J P; Ortiz, A; Pintos-Morell, G; Politei, J; Rozenfeld, P; Schiffmann, R; Svarstad, E; Talbot, A S; Thomas, M; Tøndel, C; Warnock, D; West, M L; Hughes, D A.
Afiliação
  • Moreno-Martinez D; Lysosomal Storage Disorders Unit, Royal Free Hospital NHS Foundation Trust and University College London, London, UK.
  • Aguiar P; Inborn Errors of Metabolism Reference Centre, North Lisbon Hospital Centre, Lisbon, Portugal.
  • Auray-Blais C; Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine and Health Sciences, Université de Sherbrooke, Sherbrooke, Canada.
  • Beck M; Institute of Human Genetics, University Medical Centre, University of Mainz, Mainz, Germany.
  • Bichet DG; Unité de Recherche Clinique, Centre de Recherche et Service de Néphrologie, Hôpital du Sacré-Coeur de Montreal, Montreal, Quebec, Canada.
  • Burlina A; Neurological Unit, St. Bassiano Hospital, Bassano del Grappa, Italy.
  • Cole D; Department of Medical Biochemistry and Immunology, University Hospital of Wales, Cardiff, Wales, UK.
  • Elliott P; Barts Cardiac Centre, University College London, London, UK.
  • Feldt-Rasmussen U; Medical Endocrinology and Metabolism, Rigshospitalet, Copenhagen, Denmark.
  • Feriozzi S; Division of Nephrology, Belcolle Hospital, Viterbo, Italy.
  • Fletcher J; Genetics and Molecular Pathology, SA Pathology Women's and Children's Hospital, North Adelaide, Australia.
  • Giugliani R; Medical Genetics Service, HCPA, Department of Genetics, UFRGS, Porto Alegre, Rio Grande do Sul, Brazil.
  • Jovanovic A; Department of Endocrinology and Metabolic Medicine, Salford Royal NHS Foundation Trust, Salford, UK.
  • Kampmann C; Centre for Paediatric and Adolescent Medicine, University Medical Centre, University of Mainz, Mainz, Germany.
  • Langeveld M; Department of Endocrinology and Metabolism, Academic Medical Centre, University of Amsterdam, Amsterdam, The Netherlands.
  • Lidove O; Department of Internal Medicine, Université Paris 7, Hôpital Bichat Claude-Bernard, Paris, France.
  • Linhart A; Department of Cardiovascular Medicine, First Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic.
  • Mauer M; Department of Paediatrics, University of Minnesota, Minneapolis, MN, United States.
  • Moon JC; Cardiac Imaging Department, Barts Heart Centre, London, UK.
  • Muir A; Belfast Heart Centre, Royal Victoria Hospital, Belfast, UK.
  • Nowak A; Department of Endocrinology and Clinical Nutrition, University Hospital Zurich and University of Zurich, Zurich, Switzerland.
  • Oliveira JP; Service of Medical Genetics, São João University Hospital Centre, Alameda Hernãni Monteiro, Porto, Portugal.
  • Ortiz A; Fundación Jiménez Díaz (IIS-FJD) Área de Patología Cardiovascular, Renal e Hipertensión, Madrid, Spain.
  • Pintos-Morell G; Rare and Metabolic Diseases Unit, Vall Hebron University Hospital, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Politei J; Fundation for the Study of Neurometabolic Diseases, FESEN, Argentina.
  • Rozenfeld P; Departamento de Ciencias Biológicas, CONICET, Facultad de Ciencias Exactas, IIFP, Universidad Nacional de La Plata, La Plata, Argentina.
  • Schiffmann R; Institute of Metabolic Disease, Baylor Research Institute, Dallas, TX, USA.
  • Svarstad E; Department of Clinical Medicine, University of Bergen and Haukeland University Hospital, Bergen, Norway.
  • Talbot AS; Department of Nephrology, Royal Melbourne Hospital, Melbourne, Victoria, Australia.
  • Thomas M; Department of Nephrology, Royal Perth Hospital, Perth, Western Australia, Australia.
  • Tøndel C; Clinical Trials Unit, Haukeland University Hospital, Bergen, Norway.
  • Warnock D; Division of Nephrology, University of Alabama at Birmingham, Birmingham, AL, USA.
  • West ML; Medicine, Dalhousie University, Halifax, Nova Scotia, Canada.
  • Hughes DA; Lysosomal Storage Disorders Unit, Royal Free Hospital NHS Foundation Trust and University College London, London, UK. Electronic address: derralynnhughes@nhs.net.
Mol Genet Metab ; 132(4): 234-243, 2021 04.
Article em En | MEDLINE | ID: mdl-33642210
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Ensaios Clínicos como Assunto / Doença de Fabry / Terapia de Reposição de Enzimas / Rim Tipo de estudo: Guideline Aspecto: Patient_preference Limite: Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Mol Genet Metab Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Ensaios Clínicos como Assunto / Doença de Fabry / Terapia de Reposição de Enzimas / Rim Tipo de estudo: Guideline Aspecto: Patient_preference Limite: Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Mol Genet Metab Ano de publicação: 2021 Tipo de documento: Article