Baseline characteristics at the start of nusinersen
| SMA type 1 n=7 | SMA type 2 n=12 | SMA type 3 n=9 | All SMA types n=28 | |
| Age in years at study initiation: median (range) | 0.42 (0.08–6.00) | 11.04 (4.42–18.58) | 9.08 (2.75–15.00) | 8.71 (0.08–18.58) |
| Sex—male, n (%) | 1 (14.3) | 7 (58.3) | 7 (77.8) | 15 (53.6) |
| Survival motor neuron 2 copy number | ||||
| 2 | 4 | 0 | 0 | 4 |
| 3 | 3 | 12 | 8 | 23 |
| 4 | 0 | 0 | 1 | 1 |
| Age in years at diagnosis: median (range) | 0.42 (0.08–1.50) | 1.1 (0.70–1.80) | 2.46 (1.5–12.67) | 1.17 (0.10–12.70) |
| Age in years at first nusinersen dose: median (range) | 0.42 (0.17–5.00) | 9.70 (3.60–18.80) | 7.50 (0.41–15.10) | 7.0 (0.20–18.80) |
| NIV use at the start of study: n (%) | 2 (28.6) | 8 (66.7) | 1 (11.1) | 11.0 (39.3) |
| Previous spinal instrumentation: n (%) | 0 (0.0) | 5 (41.7) | 0 (0.0) | 5 (17.9) |
NIV, non-invasive ventilation; SMA, spinal muscular atrophy.