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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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Bibliografiska uppgifter
I publikationen:Eur J Hum Genet
Huvudupphovsmän: Riccio, Eleonora, Zanfardino, Mario, Ferreri, Lucia, Santoro, Ciro, Cocozza, Sirio, Capuano, Ivana, Imbriaco, Massimo, Feriozzi, Sandro, Pisani, Antonio
Materialtyp: Artigo
Språk:Inglês
Publicerad: Springer International Publishing 2020
Ämnen:
Länkar: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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