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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...
| Autores principales: | , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer International Publishing
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7784987/ https://www.ncbi.nlm.nih.gov/pubmed/32647377 http://dx.doi.org/10.1038/s41431-020-0677-x |