Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data

The treatment options for Fabry disease (FD) are enzyme replacement therapy (ERT) with agalsidase alfa or beta, and the oral pharmacological chaperone migalastat. Since few data are available on the effects of switching from ERT to migalastat, we performed a single-center observational study on seve...

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Bibliographic Details
Published in:Eur J Hum Genet
Main Authors: Riccio, Eleonora, Zanfardino, Mario, Ferreri, Lucia, Santoro, Ciro, Cocozza, Sirio, Capuano, Ivana, Imbriaco, Massimo, Feriozzi, Sandro, Pisani, Antonio
Format: Artigo
Language:Inglês
Published: Springer International Publishing 2020
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7784987/
https://ncbi.nlm.nih.gov/pubmed/32647377
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41431-020-0677-x
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