Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

Table 2 Schedule of evaluations for α-Gal A activity (lymphocytes, peripheral blood mononuclear cells, skin and kidney) and GL-3 (plasma, 24-hour urine, skin and kidney)

W = week, α-Gal A = alpha-galactosidase A, PBMCs = Peripheral Blood Mononuclear Cells, LC/MS = Liquid Chromatography/Mass Spectrometry, GL-3 = globotriaosylceramide, LM = Light Microscopy, EM = Electron Microscopy, Q = Quantitative.
^a Multiple cell types using a subjective scoring scale of 0, 1, 2, or 3 based on increasing GL-3 content.
^b Peritubular capillaries using a modified Thurberg’s method ¹⁴.
^c Peritubular capillaries quantifying the number of GL-3 inclusion per cell using the the BLISS method ¹⁵.
^d FAB CL-202 (NCT00283959) only.
^e FAB CL-203 (NCT00283933) only.

ISSN: 1750-1172