Table 1.
Baseline characteristics of the Japanese patient population and the overall patient population of the ATTRACT study (mITT population)
| Item | Japanese patient population | Overall patient population | ||||
|---|---|---|---|---|---|---|
| Migalastat (n = 5) | ERT (n = 1) | All (n = 6) | Migalastat (n = 34) | ERT (n = 18) | All (n = 52) | |
| Age, years, mean (SD) | 51.8 (4.8) | 70.0 | 54.8 (8.6) | 51.2 (13.2) | 44.8 (14.7) | 49.0 (14.0) |
| Sex, n (%) | ||||||
| Male | 3 (60.0) | 0 | 3 (50.0) | 14 (41.2) | 8 (44.4) | 22 (42.3) |
| Female | 2 (40.0) | 1 (100.0) | 3 (50.0) | 20 (58.8) | 10 (55.6) | 30 (57.7) |
| Race, n (%) | ||||||
| White | 0 | 0 | 0 | 28 (82.4) | 17 (94.4) | 45 (86.5) |
| Asian | 5 (100.0) | 1 (100.0) | 6 (100.0) | 5 (14.7) | 1 (5.6) | 6 (11.5) |
| Other | 0 | 0 | 0 | 1 (2.9) | 0 | 1 (1.9) |
ERT enzyme replacement therapy, mITT modified intention-to-treat population; SD, standard deviation
Patients in the mITT population are those with amenable GLA mutations who were assessed for baseline and postbaseline efficacy measures