Table 4.
Eteplirsen-treated patients: Primary and secondary efficacy measures at Baseline and Week 96
| Eteplirsen-treated group | ||
| Endpoints | Baseline (n = 67) | Week 96 (n = 66) |
| 6MWT distance, m | ||
| Mean±SD | 374.6±44.1 | 256.2±148.7a |
| Range | 303.0, 449.5 | 0.0, 496.0 |
| Ability to rise independently, n (%) | 58 (86.6) | 33 (54.1)b |
| Patients with LOA, n (%) | - | 12 (17.9)c |
| NSAA total score | ||
| Mean±SD | 21.4±6.9 | 14.9±8.8b |
| Range | 4.5, 34.0 | 0.0, 34.0 |
| FVC%p, % | ||
| Mean±SD | 90.4±16.0 | 87.3±16.3 |
| Range | 50.0, 126.0 | 56.0, 128.4 |
Abbreviations: FVC%p = percent predicted forced vital capacity; LOA = loss of ambulation; 6MWT = 6-minute walk test; NSAA = North Star Ambulatory Assessment; SD = standard deviation. an = 65. bn = 61. cn = 67.