Table 1.
Baseline characteristics of the SMA type III‐treated patients according to the disease onset.
| All | IIIA | IIIB | |
|---|---|---|---|
| N | 144 | 74 | 70 |
| Sex, n (%) | |||
| Male | 84 (58.33) | 40 (54.05) | 44 (62.86) |
| Female | 60 (41.67) | 34 (45.95) | 26 (37.14) |
| Age at baseline (years), median (1st–3rd quartile) | 16.42 (9.14–35.69) | 12.60 (5.5–26.27) | 23.22 (13.07–43.94) |
| Age < 18 years, n (%) | 77 (53.47) | 51 (68.92) | 26 (37.14) |
| Median age in pediatric population (1st–3rd quartile), years | 9.50 (5.50–13.43) | 8.01 (4.40–13.11) | 11.74 (9.24–15.08) |
| Age ≥ 18 years, n (%) | 67 (46.53) | 23 (31.08) | 44 (62.86) |
| Median age in adult population (1st–3rd quartile), years | 36.60 (26.27–47.08) | 35.40 (27.10–39.00) | 38.84 (25.51–49.35) |
| Disease duration (years), median (1st–3rd quartile) | 12.10 (4.4–28.89) | 10.41 (3.51–25.11) | 13.33 (4.66–31.43) |
| SMN2 copy number, n (%) | |||
| 1 | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| 2 | 11 (7.64) | 4 (5.41) | 7 (10.00) |
| 3 | 56 (38.88) | 40 (54.05) | 16 (22.85) |
| 4 | 14 (9.72) | 8 (10.81) | 6 (8.57) |
| 4+ | 29 (20.14) | 6 (8.11) | 23 (32.86) |
| Unknown | 34 (23.61) | 16 (21.62) | 18 (25.71) |
| SMA function, n (%) | |||
| Non‐sitter | 3 (2.08) | 3 (4.05) | 0 (0.00) |
| Sitter | 62 (43.06) | 40 (54.05) | 22 (31.43) |
| Walker | 79 (54.86) | 31 (41.89) | 48 (68.57) |
| Baseline HFMSE score, median (1st–3rd quartile) |
41 (23–54) N = 130 |
32.50 (15–50) N = 66 |
48.5 (28.0–58.5) N = 64 |
| Baseline RULM score, median (1st–3rd quartile) |
31 (24–37) N = 116 |
27 (22–32) N = 56 |
35.5 (29.5–37.0) N = 60 |
| Baseline 6MWT meter, median (1st–3rd quartile) |
321.5 (166–425) N = 62 |
283 (107–397) N = 23 |
356 (236–434) N = 39 |
| Follow‐up (years), mean (SD) | 1.83 (0.61) | 1.91 (0.63) | 1.75 (0.58) |
| No of visits, median (range) | 5 (2–11) | 6 (2–11) | 5 (3–11) |