Table 1.
Demographic characteristics of SMA patients at baseline.
| Distribution of patients-SMA type; n (%) | 310 (100) |
| Type 1a | 156 (50.3) |
| Type 1b | 120 (38.7) |
| Type 1c | 34 (11) |
| SMN copies; n (%) | |
| 2 copies | 302 (97.4) |
| 3 copies | 8 (2.6) |
| Gender | |
| Female; n (%) | 162 (52.3) |
| Male; n (%) | 148 (47.7) |
| Age at onset of SMA; mean + SD (range) in months | 2.4 ± 1.1 (0–5) |
| Age at onset of treatment, median (range) in months | 5 (1–120) |
| Monitoring time, median (range) in months | 14 (2–48) |
| CHOP-INTEND score, at baseline; median (range) | 16 (0–50) |
| CHOP-INTEND score, post-treatment; median (range) | 28.0.5 (1–64) |
| Number of Nusinersen doses, median, (range) | 5 (4–15) |
| Events of death during monitoring; n (%) | 66 (21.3) |
| First year (%, within death) | (68.2) |
| Second year (%, within death) | (27.3) |
| Third year (%, within death) | (6.1) |
| Presymptomatic patients n (%) | 7 (2.2) |