Migalastat is an oral chaperone therapy for patients diagnosed with Fabry disease and who have an amenable variant.1 The mechanism of action of migalastat is presented in Figure 1. The availability of migalastat differs between countries. For further information, please consult your local prescribing information.
Mechanism of action of migalastat.1,2
C-ANPROM/INT/FAB/0017; Date of preparation: March 2021
- Amicus Therapeutics Europe Ltd. Galafold® EU Summary of Product Characteristics. Last updated August 2020.
- Ferraz MJ, Kallemeijn WW, Mirzaian M, et al. Gaucher disease and Fabry disease: new markers and insights in pathophysiology for two distinct glycosphingolipidoses. Biochim Biophys Acta 2014; 1841: 811-825.