Table 4.
Ambulatory and Pulmonary Function in Golodirsen-Treated Patients
| Endpoints | Baselinea | Week 48 | Week 96 | Week 144 |
|---|---|---|---|---|
| 6MWT, m | n = 25 | n = 23 | n = 24 | n = 22 |
| Mean (SD) | 405.8 (55.1) | 378.9 (93.2) | 344.1 (128.9) | 311.0 (143.4) |
| Range | 290 − 512 | 81 − 541 | 0 − 523 | 0 − 481 |
| Mean (SD) change from baseline | −26.1 (65.1) | −64.6 (105.1) | −99.0 (123.8) | |
| Loss of ambulation | n = 25 | n = 25 | n = 24 | n = 23 |
| n (%) | 0 | 0 | 1 (4.0) | 2 (9.0) |
| FVC%p | n = 25 | n = 24 | n = 23 | n = 23 |
| Mean (SD) | 92.7 (24.0) | 92.5 (18.7) | 93.9 (17.6) | 83.8 (23.2) |
| Range | 16.4–137.8 | 41.9–129.9 | 37.4–124.9 | 7.8–121.1 |
| Mean (SD) change from baseline | −0.63 (21.5) | 0.79 (23.8) | −8.38 (29.5) |
a
Baseline FVC%p for placebo patients was defined as Part 2 baseline FVC%p, and inclusion criteria (FVC%p > 50%) for screening were not applied at that time.