Table 3 Change in plasma lyso-Gb3 and Gb3 levels (efficacy population)
Parameter | Men n = 13 | Women n = 7 | Overall N = 20 | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Month 12 | Change from baseline | % change | Baseline | Month 12 | Change from baseline | % change | Baseline | Month 12 | Change from baseline | % change | |
Plasma lyso-Gb3 level, nmol/L | ||||||||||||
Mean (SD)a | 51.8 (49.0) | 32.3 (24.9) | − 19.6 (27.2) | − 32.4 (4.8) | 13.8 (6.1) | 9.2 (2.9) | − 4.6 (3.8) | − 29.8 (4.7) | 38.5 (43.3) | 24.2 (22.8) | − 14.3 (23.0) | − 31.5 (3.5) |
Median (range) | 42.4 (1, 189) | 29.0 (1, 90) | − 8.2 (− 99, 4) | − 36.1 (− 53, 9) | 12.9 (7, 23) | 10.6 (5, 13) | − 2.7 (− 11, − 1) | − 23.3 (− 46, − 17) | 22.1 (1, 189) | 13.4 (1, 90) | − 6.6 (− 99, 4) | − 34.5 (− 53, 9) |
Plasma Gb3 level, μmol/L | ||||||||||||
Mean (SD)a | 6.4 (2.0) | 5.6 (1.4) | − 0.8 (1.5) | − 9.0 (5.6) | 5.5 (1.9) | 4.7 (1.2) | − 0.8 (1.1) | − 11.2 (6.9) | 6.1 (2.0) | 5.3 (1.4) | − 0.8 (1.3) | − 9.8 (4.3) |
Median (range) | 6.2 (3, 11) | 5.5 (3, 8) | 0.04 (− 4, 1) | 0.6 (− 40, 19) | 6.1 (3, 8) | 4.8 (2, 6) | − 0.8 (− 3, 1) | − 18.4 (− 32, 18) | 6.2 (3, 11) | 5.3 (2, 8) | − 0.7 (− 4, 1) | − 13.7 (− 40, 19) |