Current standard of care for the treatment of Fabry disease consists of adjunctive therapies for management of symptoms and complications, and chronic/life-long disease-modifying therapies aimed at increasing the availability of functional lysosomal α-Gal A. Approved disease-modifying therapies for Fabry include enzyme replacement therapy (ERT) with intravenous recombinant or gene-activated α-Gal A (agalsidase-alfa or agalsidase-beta) and oral pharmacological chaperone therapy (PCT) with migalastat [2]. The gene discussed is GLA; the disease is Fabry disease.