Interní Med. 2017; 19(3): 167-170 | DOI: 10.36290/int.2017.028
Migalastat in the treatment of Fabry disease
- II. interní klinika VFN a 1. LF UK, Praha
Fabry disease is an inherited disease with multiorgan involvement. Until now the only specific therapy was intravenous enzyme
replacement. In a short time a new oral therapy with migalastat is expected on our market. The medication is indicated only for
treatment of certain mutations with residual enzyme activity. It binds to the enzyme, stabilizes it and enhances its transportation
to lysosomes. Results of clinical studies are favourable and indicate that the new medication migalastat could significantly increase
the quality of care of selected patients.
Keywords: Fabry disease, specific therapy, enzyme-replacement therapy, migalastat
Published: July 1, 2017 Show citation
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