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

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

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 chang...

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Bibliographic Details
Main Authors: Daniel G. Bichet, Roser Torra, Eric Wallace, Derralynn Hughes, Roberto Giugliani, Nina Skuban, Eva Krusinska, Ulla Feldt-Rasmussen, Raphael Schiffmann, Kathy Nicholls
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Classic phenotype
Efficacy
Fabry disease
Chaperone
Migalastat
Renal function
Online Access:http://www.sciencedirect.com/science/article/pii/S221442692100080X

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http://www.sciencedirect.com/science/article/pii/S221442692100080X

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