Table 1 Baseline demographic characteristics, baseline motor function and pre-treatment characteristics
Baseline characteristics | Enrolled patients | Patients treated with risdiplam | ||
|---|---|---|---|---|
SMA1 | SMA2 | SMA1 | SMA2 | |
Total number of patients | 36 | 98 | 31 | 80 |
Female/male, n (%) | 16/20 (44.4/55.6) | 59/39 (60.2/39.8) | 14/17 (45.2/54.8) | 49/31 (61.3/38.8) |
Mean ± SD age at start of enrollment, years | 13.1 ± 10.4 | 27.6 ± 14.1 | 13.5 ± 11.1 | 29.0 ± 14.0 |
Mean ± SD age at SMA onset, years | 2.8 ± 2.3 | 11.3 ± 4.1 | 2.65 ± 2.18 | 11.1 ± 4.4 |
Mean ± SD weight, kg | 27.3 ± 11.2* | 46.4 ± 19.0 | 25.8 ± 9.84* | 46.3 ± 19.4 |
Prior therapies | ||||
Nusinersen, n (%) | 28 (77.8) | 51 (52.0) | 23 (74.2) | 37 (46.3) |
Mean ± SD nusinersen treatment duration, months | 31.9 ± 16.6 | 25.7 ± 11.2* | 28.6 ± 15.7 | 23.9 ± 11.3 |
Onasemnogene abeparvovec, n (%) | 0 | 0 | 0 | 0 |
No SMA pre-treatment, n (%) | 8 (22.2) | 47 (48.0) | 8 (25.8) | 43 (53.8) |
Baseline motor function, n (%) Current level of function/highest motor function achieved | ||||
Supports head unaided | 6 (16.7)/10 (27.8) | 33 (33.7)/7 (7.1) | 5 (16.1)/7 (22.6) | 22 (27.5)/5 (6.3) |
Sitting unaided | 0/0 | 41 (41.8)/66 (67.3) | 0/0 | 36 (45.0)/53 (66.3) |
Crawls on stomach | 0/0 | 3 (3.1)/17 (24.4) | 0/0 | 3 (3.8)/15 (18.8) |
Stands unaided | 0/0 | 0/7 (7.1) | 0/0 | 0/6 (7.5) |
Walks unaided | 0/0 | 0/0 | 0/0 | 0/0 |
None of the above | 30 (83.3)/26 (72.2) | 21 (21.4)/1 (1.0) | 26 (83.9)/24 (77.4) | 19 (23.8)/1 (1.3) |