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.
What are the chaperone therapies for Fabry disease?
One oral chaperone therapy, migalastat, is available for patients diagnosed with Fabry disease and who have an amenable variant.
What is agalsidase alfa?
Agalsidase alfa is an intravenous enzyme replacement therapy for patients with Fabry disease.
What is agalsidase beta?
Agalsidase beta is an intravenous enzyme replacement therapy for patients with Fabry disease.
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