Prakt. Lékáren. 2017; 13(4e) [Interní Med. 2017; 19(3): 167-170]

Migalastat in the treatment of Fabry disease

Lubor Goláň
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 mutationswith residual enzyme activity. It binds to the enzyme, stabilizes it and enhances its transportation to lysosomes. Results of clinical studiesare 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: December 1, 2017  Show citation

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Goláň L. Migalastat in the treatment of Fabry disease. Praktické lékárenství. 2017;13(E-verze 4/17):.
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