Table 1.
ID | Sex | Earlier Treatment (Age in Years), Type of Treatment | Onasemnogene Abeparvovec Age (Years) | SMA Type | SMN2 Gene Copies | Ambulation Status at the Time of Imaging | Age at Imaging (Years) |
---|---|---|---|---|---|---|---|
1 | F | 0.2, Nusinersen | 0.2 | 1 | 2 | non-ambulatory | 1.6 |
2 | F | 0.1, Nusinersen | 0.2 | 1 | 2 | non-ambulatory | 1.4 |
3 | M | 0.2, Risdiplam | 0.3 | 1 | 2 | non-ambulatory | 1.1 |
4 | F | 0.3 | 1 | 2 | non-ambulatory | 1.5 | |
5 | F | 0.7 | 1 | 2 | non-ambulatory | 1.6 | |
6 | F | 0.5, Nusinersen | 0.9 | 1 | 2 | non-ambulatory | 2.2 |
7 | M | 0.5, Nusinersem | 1.3 | 1 | 2 | non-ambulatory | 2.3 |
8 | M | 0.4, Nusinersen | 1.3 | 1 | 2 | non-ambulatory | 3.7 |
9 | M | 0.3, Nusinersen | 1.4 | 1 | 2 | non-ambulatory | 3.5 |
10 | M | 1.5 | 2 | 3 | non-ambulatory | 3.6 | |
11 | M | 1.3, Nusinersen | 1.5 | 2 | 3 | minimal ambulatory | 4.0 |
12 | M | -, Risdiplam | 1.5 | 1 | 3 | ambulatory | 1.6 |
13 | M | 0.2, Nusinersen | 1.7 | 1 | 2 | non-ambulatory | 5.6 |
14 | M | 1.6, Nusinersen | 3.3 | 2 | 3 | non-ambulatory | 4.8 |
15 | F | 3.7 | 1 | 2 | non-ambulatory | 4.3 | |
16 | F | 4.9 | 2 | 3 | non-ambulatory | 6.9 |