Long-term follow-up of renal function in patients treated with migalastat for Fabry disease.

Bichet, Daniel G; Torra, Roser; Wallace, Eric; Hughes, Derralynn; Giugliani, Roberto; Skuban, Nina; Krusinska, Eva; Feldt-Rasmussen, Ulla; Schiffmann, Raphael; Nicholls, Kathy

Bichet, Daniel G; Torra, Roser; Wallace, Eric; Hughes, Derralynn; Giugliani, Roberto; Skuban, Nina; Krusinska, Eva; Feldt-Rasmussen, Ulla; Schiffmann, Raphael; Nicholls, Kathy.

Afiliação

Bichet DG; Department of Medicine, Hôpital du Sacré-Coeur, University of Montréal, Montreal, Quebec, Canada.
Torra R; Inherited Renal Disorders, Nephrology Department, Fundació Puigvert, REDINREN, IIB Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.
Wallace E; Department of Medicine, University of Alabama, Birmingham, AL, USA.
Hughes D; Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust and University College London, London, UK.
Giugliani R; Medical Genetics Service, Department of Genetics, Institute of Biosciences, Institute of Basic Health Sciences, Faculty of Medicine, Faculty of Pharmacy (UFRGS) and, National Institute of Population Medical Genetics (INAGEMP), Porto Alegre, Brazil.
Skuban N; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Krusinska E; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Feldt-Rasmussen U; Department of Medical Endocrinology and Metabolism, Rigshospitalet, National University Hospital, Copenhagen University, Copenhagen, Denmark.
Schiffmann R; Institute of Metabolic Disease, Baylor Scott & White Research Institute, Dallas, TX, USA.
Nicholls K; Department of Nephrology, Royal Melbourne Hospital, University of Melbourne, Parkville, Victoria, Australia.

Mol Genet Metab Rep ; 28: 100786, 2021 Sep.

Article em En | MEDLINE | ID: mdl-34401344

Palavras-chave

Chaperone; Classic phenotype; Efficacy; Fabry disease; GLP-HEK, Good Laboratory Practice-validated human embryonic kidney; Gb3, globotriaosylceramide; LVMi, left ventricular mass index; Migalastat; Q1, quartile 1; Q3, quartile 3; RI, renin inhibitor; Renal function; eGFRCKD-EPI, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation; α-Gal A, α-galactosidase A

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Genet Metab Rep Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Canadá

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