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Long-term follow-up of renal function in patients treated with migalastat for Fabry disease / Bichet, Daniel G. (Department of Medicine, Hôpital du Sacré-Coeur, University of Montréal, Montreal, Quebec, Canada) ; Torra Balcells, Roser (Institut d'Investigació Biomèdica Sant Pau) ; Wallace, Eric (Department of Medicine, University of Alabama, Birmingham, AL, USA) ; Hughes, Derralynn A (Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust and University College London, London, UK) ; Giugliani, Roberto (Instituto Nacional de Genética Médica Populacional (Porto Alegre, Brasil)) ; Skuban, Nina (Amicus Therapeutics, Inc., Cranbury, NJ, USA) ; Krusinska, Eva (Amicus Therapeutics, Inc., Cranbury, NJ, USA) ; Feldt-Rasmussen, Ulla (Copenhagen University Hospital Rigshospitalet) ; Schiffmann, Raphael (Institute of Metabolic Disease, Baylor Scott & White Research Institute, Dallas, TX, USA) ; Nicholls, Kathy (Royal Melbourne Hospital (Melbourne, Austràlia)) ; Universitat Autònoma de Barcelona
The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term changes in renal function in patients with Fabry disease and amenable GLA variants who were treated with migalastat for ≥2 years during these studies. [...]
2021 - DOI (Digital Object Identifier)

10.1016/j.ymgmr.2021.100786
Molecular Genetics and Metabolism Reports, Vol. 28 (august 2021)

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