Table 1.
Baseline characteristics in the CS2 study
| Characteristic | Total cohorta (N = 14) | By age group at screening | |
|---|---|---|---|
| ≤11 years (n = 9) | >11 years (n = 5) | ||
| Male, n (%) | 6 (43) | 4 (44) | 2 (40) |
| Age at symptom onset, months | |||
| Mean (SD) | 23.9 (13.6) | 18.2 (7.3) | 34.2 (16.9) |
| Median (range) | 18.0 (11–60) | 18.0 (11–36) | 36.0 (15–60) |
| Age at SMA diagnosis, months | |||
| Mean (SD) | 47.4 (34.6) | 36.7 (23.9) | 66.6 (44.9) |
| Median (range) | 33.5 (15–144) | 27.0 (15–96) | 56.0 (29–144) |
| SMN2 copy number, n (%) | |||
| 3 copies | 9 (64) | 7 (78) | 2 (40) |
| 4 copies | 5 (36) | 2 (22) | 3 (60) |
| SMA type, n (%) | |||
| Type II | 1 (7) | 1 (11) | 0 |
| Type III | 13 (93) | 8 (89) | 5 (100) |
| Ambulatory, n (%) | 13 (93) | 8 (89) | 5 (100) |
| Nonambulatory, n (%) | 1 (7) | 1 (11) | 0 |
| Dose level in CS2, n (%) | |||
| 3 mg | 2 (14) | 0 | 2 (40) |
| 6 mg | 0 | 0 | 0 |
| 9 mg | 6 (43) | 5 (56) | 1 (20) |
| 12 mg | 6 (43) | 4 (44) | 2 (40) |
| Time from end of CS2 to first dose in CS12, days | |||
| Mean (SD) | 119.3 (79.9) | 113.0 (69.8) | 129.4 (102.1) |
| Median (range) | 118.0 (19–233) | 104.0 (26–196) | 168.0 (19–233) |
Abbreviations: SMA, spinal muscular atrophy; SMN, survival motor neuron.
a
All children who received their first nusinersen dose in the CS2 study, were ambulatory during CS2 or CS12, and performed the 6MWT were included in these analyses.