Table 2 Baseline characteristic of 31 patients
Parameter | |
|---|---|
Age, years, mean ± SD | 52.4 ± 15.1 |
Sex, n (%) | |
Female | 16 (51.6) |
Male | 15 (48.4) |
Classic Fabry disease, n (%) | 23 (74.2) |
Plasma Lyso-Gb3, mean ± SD (range), nMol/L (normal value < 1.9) | 33.7 ± 31.0 (3.0–115.4) |
Serum α-Gal A activity, mean ± SD (range), nMol/h/mL (reference range > 3.8) | 0.13 ± 1.1 (0.0–0.61) |
Late-onset disease, n (%) | 8 (25.8) |
Plasma Lyso-Gb3, mean ± SD (range), nMol/L (normal value < 1.9) | 3.6 ± 4.0 (1.5–22.3) |
Serum α-Gal A activity, mean ± SD (range) nMol/h/mL (reference range > 3.8) | 3.8 ± 6.7 (0.10–16.3) |
Prior therapy, n (%) | |
Naïve | 25 (80.6) |
Agalsidase alfa | 6 (19.4) |