Fig. 2: α-galactosidase A (α-gal A) enzyme activity and vector copy number (VCN).

a Plasma α-gal A activity attained reference range levels in all patients; although decreased over time, the plasma α-gal A enzyme activity levels are above what is observed in Fabry disease patients and have not returned to original baseline levels. The reference ranges (dotted lines) were defined by Dr. Rupar’s laboratory based on 150 specimens referred for diagnostic testing. Males with classic Fabry disease have plasma levels around 1 nmol/h/ml. b Leukocyte α-gal A attained supranormal specific activity levels for each patient. Although decreased over time, leukocyte α-gal A-specific enzyme activity levels are above what is seen in Fabry disease patients and have not returned to original baseline levels. The reference ranges (dotted lines) were defined by Dr. Rupar’s laboratory based on 150 specimens referred for diagnostic testing. c VCN in peripheral blood reached between 0.55 and 1.10 copies/genome in all patients, and although decreased over time, has remained above 0.05 copies/genome in all patients to date (almost 3 years in Patient 1).