In Vitro and In Vivo Amenability to Migalastat in Fabry Disease
Migalastat (1-deoxygalactonojirimycin) is approved for the treatment of Fabry disease (FD) in patients with an amenable mutation. Currently, there are at least 367 amenable and 711 non-amenable mutations known, based on an in vitro good laboratory practice (GLP) assay. Recent studies demonstrated th...
Main Authors: | , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2020-12-01
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Series: | Molecular Therapy: Methods & Clinical Development |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050120301789 |