Table 1.
Summary of patient demographics and treatment data
| SMA type | SMN2 copy number | Nusinersen treatment initiation | Onasemnogene Abeparvovec treatment | last follow-up | |||
|---|---|---|---|---|---|---|---|
| age [d] | age [d] | body weight [kg] | nutritional and/or ventilatory support; scoliosis | age [d] | nutritional an/or ventilatory support; scoliosis | ||
| 1b | 2 | 39 | 102 | 6,4 | – | 606 | S |
| 1a | 2 | 80 | 312 | 7,6 | N (gs) | 933 | N (gs), S |
| 1b | 2 | – | 75 | 5,3 | – | 502 | S |
| 1b | 2 | – | 97 | 6,0 | – | 518 | N (gs), S |
| 1b | 2 | – | 79 | 4,8 | – | 494 | N (ng), V |
| 1b | 2 | – | 192 | 5,8 | – | 374 | – |
| 2 | 3 SMN hybrid gene copies | – | 527 | 8,7 | – | 830 | – |
| ps | 3 | – | 19 | 4,5 | – | 286 | – |
| ps | 3 SMN hybrid gene copies | – | 37 | 4,0 | – | 340 | – |
ps pre-symptomatic, N nutritional support (gs gastrostomy, ng nasogastric tube), V ventilatory support, S scoliosis