For US healthcare professionals only
Small-molecule chaperones are oral treatments designed to bind active sites of certain mutant forms of the alpha-galactosidase A (alpha-Gal A) enzyme, stabilize them, and help facilitate proper trafficking of the endogenous alpha-Gal A to lysosomes.1
ERTs are infusion treatments designed to replace the patient’s endogenous enzyme with recombinant enzyme.2 For treating Fabry disease, ERT replaces the missing or deficient alpha-Gal A enzyme.2
References: 1. Germain DP. Fabry disease. Orphanet J Rare Dis. 2010;5:30. 2. Ries M. Enzyme replacement therapy and beyond—in memoriam Roscoe O. Brady, M.D. (1923–2016). J Inherit Metab Dis. 2017;40(3):343-356.