Table 1 Baseline demographics of enrolled patients, divided in patients who were enrolled treatment-naïve and during ongoing treatment.
All (n = 22) | Treatment-naïve (n = 15) | Later enrolled (n = 7) | |
|---|---|---|---|
Sex | |||
Female—n (%) | 9 (41%) | 6 (40%) | 3 (43%) |
Male—n (%) | 13 (59%) | 9 (60%) | 4 (57%) |
Age at symptom onset—years, median (range) | 2 (0.5–47.2) | 3 (0.5–47.2) | 1 (0.6–8.0) |
Age at treatment initiation—years, median (range) | 34 (19–64) | 34 (19–64) | 37 (20–63) |
SMA type—n (%) | |||
2 | 9 (41%) | 5 (33%) | 4 (57%) |
3a | 4 (18%) | 2 (13%) | 2 (29%) |
3b | 8 (36%) | 7 (47%) | 1 (14%) |
4 | 1 (5%) | 1 (7%) | 0 (0%) |
SMN2 copy number—n (%) | |||
2 | 2 (9%) | 0 (0%) | 2 (29%) |
3 | 8 (36%) | 5 (33%) | 3 (43%) |
4 | 9 (41%) | 8 (53%) | 1 (14%) |
≥ 5 | 3 (14%) | 2 (13%) | 1 (14%) |
Ambulatory status | |||
Non-ambulant—n (%) | 12 (55%) | 7 (47%) | 5 (71%) |
Ambulant—n (%) | 10 (45%) | 8 (53%) | 2 (29%) |
Scoliosis | 9 (41%) | 5 (33%) | 4 (57%) |
NIV—n (%) | 6 (27%) | 4 (27%) | 2 (29%) |
PEG—n (%) | 2 (9%) | 1 (7%) | 1 (14%) |
