Table 1 Patients’ characteristics
|  | SMA type 1 (n = 1) | SMA type 2 (n = 47) | SMA type 3 (n = 57) | SMA type 4 (n = 2) |
|---|---|---|---|---|
Mean age in years (range) | 44 | 33.3 (20–55) | 37.4 (16–62) | 48 (40–56) |
Mean age at onset (y) | NA | NA | 7.2 | 29.5 |
Mean SMN2 copy numbers | MD | 2.9 | 3.3 | 4 |
Wheel chair bound (%) | 1 (100) | 47 (100) | 31 (54) | 0 (0) |
Feeding tube | 1 | 3 | 1 | 0 |
Spine fusion surgery (%) | 1 (100) | 41 (87.2) | 18 (31.6) | 0 |
Noninvasive ventilation/invasive ventilation | 0/1 | 22/10 | 7/2 | 0/0 |
Median MFM32 (% total score) | NA | 12 (0–40.6) | 56.0 (14.6–83.0) | 83 (MD) |
Median 6MWT (meters) (range) | NA | NA | 215 (0–450) | 400 |
Median 10mWT (s) (range) | NA | NA | 9.0 (0–36) | MD |
Median RULM (range) | NA | 7 (0–18) | 17 (2–37) | MD |
Median FVC (%) (range) | NA | 25 (1.1–54) | 93 (46–140) | MD |