Table 3.
Percentages of patients grouped by SMN2 copy number reaching real-world endpoints (i.e., head holding, sitting 30 s unassisted, standing a few seconds with aid, or need for gastrostomy, NIV, or spinal brace) at M0 and M12 for all patients with 12 months of follow-up (2 deceased patients were excluded) [n = 27] (left column) and at M0, M12 and M24 for patients with 24 months of follow-up (2 deceased patients were excluded) [n = 17] (right)
| SMN2 copies | 12 months follow up | 24 months follow up | ||
|---|---|---|---|---|
| 2 | 3 | 2 | 3 | |
| N = 20 | N = 7 | N = 13 | N = 4 | |
| Head holding | ||||
| M0 | 45 | 86 | 31 | 75 |
| M12 | 95 | 100 | 92 | 100 |
| M24 | – | – | 100 | 100 |
| Sitting 30s | ||||
| M0 | 0 | 0 | 0 | 0 |
| M12 | 50 | 100 | 54 | 100 |
| M24 | – | – | 85 | 100 |
| Standing with aid | ||||
| M0 | 0 | 0 | 0 | 0 |
| M12 | 10 | 14 | 8 | 0 |
| M24 | – | – | 62 | 100 |
| VNI | ||||
| M0 | 5 | 0 | 8 | 0 |
| M12 | 30 | 0 | 23 | 0 |
| M24 | – | – | 23 | 0 |
| Gastrostomy | ||||
| M0 | 0 | 0 | 0 | 0 |
| M12 | 10 | 0 | 8 | 0 |
| M24 | – | – | 15 | 0 |
| Spinal brace | ||||
| M0 | 0 | 0 | 0 | 0 |
| M12 | 50 | 14 | 54 | 0 |
| M24 | – | – | 85 | 100 |
All reported numbers above are percentages (%)