![](/img/cover-not-exists.png)
The effects of long-term migalastat treatment in Fabry disease patients previously treated with enzyme replacement therapy who have migalastat-amenable variants with low alpha-galactosidase A response in the in vitro migalastat amenability assay
Nicholls, Kathleen, Olivotto, Iacopo, Ohashi, Toya, Williams, Hadis, Jain, Vipul, Skuban, NinaVolume:
126
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2018.12.272
Date:
February, 2019
File:
PDF, 80 KB
2019