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

Autor: Bichet, Daniel G., Torra Balcells, Roser, Wallace, Eric, Hughes, Derralynn A, Giugliani, Roberto, Skuban, Nina, Krusinska, Eva, Feldt-Rasmussen, Ulla, Schiffmann, Raphael, Nicholls, Kathy, Universitat Autònoma de Barcelona

Publikováno v: Mol Genet Metab Rep
Molecular Genetics and Metabolism Reports
r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau
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Molecular Genetics and Metabolism Reports, Vol 28, Iss, Pp 100786-(2021)
Repositório Institucional da UFRGS
Universidade Federal do Rio Grande do Sul (UFRGS)
instacron:UFRGS
Dipòsit Digital de Documents de la UAB
Universitat Autònoma de Barcelona
Bichet, D G, Torra, R, Wallace, E, Hughes, D, Giugliani, R, Skuban, N, Krusinska, E, Feldt-Rasmussen, U, Schiffmann, R & Nicholls, K 2021, ' Long-term follow-up of renal function in patients treated with migalastat for Fabry disease ', Molecular Genetics and Metabolism Reports, vol. 28, 100786 . https://doi.org/10.1016/j.ymgmr.2021.100786

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 extensi

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https://ddd.uab.cat/record/248892