Table 2.
Summary of adverse events
| ERT experienceda (n = 23) | ERT naïve (n = 6) | Overall (N = 29) | |
|---|---|---|---|
| Patients with any TEAE, n (%) | 23 (100) | 6 (100) | 29 (100) |
| TEAEs potentially related to treatment | 16 (70) | 4 (67) | 20 (69) |
| Serious TEAEs | 8 (35) | 4 (67) | 12 (41) |
| Serious TEAEs potentially related to treatment | 2 (9) | 2 (33) | 4 (14) |
| TEAEs leading to study withdrawal | 2 (9) | 0 (0) | 2 (7) |
| Severe TEAEs | 6 (26) | 3 (50) | 9 (31) |
| TEAEs leading to death | 0 (0) | 0 (0) | 0 (0) |
| IARs | 10 (43) | 3 (50) | 13 (45) |
ERT enzyme replacement therapy, IAR infusion-associated reaction, TEAE treatment-emergent adverse event with onset date on or after the first dose of study drug
aPooled data for ERT-experienced cohorts 1, 2 and 4 (includes both ambulatory and non-ambulatory patients)