Patients with SMA (n = 28) | |
---|---|
Age, y, mean (SD) | 27.8 (10) |
Age of onset, y, mean (SD) | 7.81 (9.91) |
Course of the disease, y, mean (SD) | 19.97 (10.04) |
Sex, F/M | 11/17 |
SMA type (2/3/4), n | 8/17/3 |
SMN2 copies (2/3/≥4), n (%) | 3/15/10 |
Motor performance (non-sitter/sitter/ambulant), n | 5/8/15 |
Baseline HFMSE, mean (SD) | 31.7 (23.9) |
FVC, % (SD) | 79.7 (33.9) |
Creatinine, umol/L (SD) | 23.7 (14.8) |