TABLE 3.
Baseline assessments of patients treated with delandistrogene moxeparvovec compared with matched, propensity-score-weighted EC cohorts for functional endpoints A) Part 1.
| Baseline parameter | Delandistrogene moxeparvovec | EC |
|---|---|---|
| (N = 19) | (N = 51) | |
| Age (years) | ||
| Mean (SD) | 6.21 (1.17) | 6.20 (0.45) |
| Median | 6.49 | 6.10 |
| NSAA total score | ||
| Mean (SD) | 19.9 (3.4) | 19.7 (1.9) |
| Median | 20.0 | 20.0 |
| Time to Rise (seconds) | ||
| Mean (SD) | 5.17 (2.21) | 5.22 (1.05) |
| Median | 4.60 | 4.70 |
| 10MWR (seconds) | ||
| Mean (SD) | 5.39 (1.16) | 5.39 (0.58) |
| Median | 5.10 | 5.50 |
| B) Part 2 (crossover) | ||
| Baseline parameter | Delandistrogene moxeparvovec | EC |
|---|---|---|
| (N = 20) | (N = 103) | |
| Age (years) | ||
| Mean (SD) | 7.24 (1.12) | 7.03 (0.42) |
| Median | 7.07 | 6.97 |
| NSAA total score | ||
| Mean (SD) | 23.8 (3.7) | 23.5 (1.9) |
| Median | 24.5 | 24.0 |
| Time to Rise (seconds) | ||
| Mean (SD) | 4.02 (1.34) | 3.92 (0.59) |
| Median | 3.80 | 3.70 |
| 10MWR (seconds) | ||
| Mean (SD) | 4.84 (1.15) | 4.83 (0.40) |
| Median | 4.65 | 4.90 |
10MWR, 10-m Walk/Run; EC, external control; NSAA, North Star Ambulatory Assessment; SD, standard deviation.