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Table 1 Baseline demographic characteristics, baseline motor function and pre-treatment characteristics

From: Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

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)
  1. *2 patients with missing data
  2. SD standard deviation, SMA1 spinal muscular atrophy type 1, SMA2 spinal muscular atrophy type 2