Folic acid supplementation for treatment of fabry disease

Abstract

A therapeutic approach for treating patients with Fabry disease by supplementing or co-administration of synthetic folic acid (FA) to substantially stimulate α-galactosidase (AGLA) activity in combination with another therapy. Stimulation of AGLA activity may help Fabry disease patients on different types of primary therapeutic regiments manage clinical symptoms with a reduced dose of the primary treatment medication. Folic acid may thus be co-administered with an enzyme replacement therapy, such as recombinant α-galactosidase A, or a chaperone therapy, such as specific inhibitor, 1-deoxygalactonojiromycin, to improve the efficacy of the treatment.

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