Table 3.
Change from baseline in functional endpoints
| Mean (SD) change from baseline | TAZPOWER patients (n = 8) | Natural history controls (n = 19) |
|---|---|---|
| 6MWT, meters | ||
| LS mean change from baseline at Week 64 | 80.299 | 0.596 |
| LS mean difference (95% CI); P value | 79.703 (40.46, 118.95); P = 0.0004 | |
| LS mean change from baseline at Week 76 | 91.858 | 0.886 |
| LS mean difference (95% CI); P value | 90.972 (44.57, 137.38); P = 0.0005 | |
| Muscle strength by HHD | ||
| LS mean change from baseline at Week 64 | 41.789 | 1.035 |
| LS mean difference (95% CI); P value | 40.755 (21.70, 5980); P = 0.0002 | |
| LS mean change from baseline at Week 76 | 48.667 | 1.970 |
| LS mean difference (95% CI); P value | 40.755 (21.70, 59.80); P = 0.0005 | |
| 5XSST | ||
| LS Mean change from baseline at Week 64 | − 2.361 | − 0.002 |
| LS mean difference (95% CI); P value | − 2.359 (− 4.62, − 0.10); P = 0.0416 | |
| LS mean change from baseline at Week 76 | − 2.829 | − 0.003 |
| LS mean difference (95% CI); P value | − 2.825 (− 5.41, − 0.24): P = 0.0340 | |
| SWAY application balance assessment | ||
| LS Mean change from baseline at Week 64 | 7.398 | 0.862 |
| LS mean difference (95% CI); P value | 6.536 (− 2.21, 15.28); P = 0.1312 | |
| LS mean change from baseline at Week 76 | 8.806 | 1.084 |
| LS mean difference (95% CI); P value | 7.722 (− 2.40, 17.84); P = 0.1240 | |
| MDRI | ||
| LS Mean change from baseline at Week 64 | 3.013 | 0.634 |
| LS mean difference (95% CI); P value | 2.379 (1.34, 3.42); P = 0.0001 | |
| LS mean change from baseline at Week 76 | 3.119 | 0.709 |
| LS mean difference (95% CI); P value | 2.411 (1.38, 3.44); P = 0.0001 | |
6MWT 6-min walk test, HHD Handheld dynamometry, MDRI Multi-domain responder index, 5XSST 5 times sit-to-stand, SD Standard deviation