Table 1.

Demographics and clinical data of patients with CT-guided nusinersen treatment.

	SMA type 2	SMA type 3	Total
Number of patients	6 (54.5%)	5 (45.5%)	11 (100%)
Demographic data
Mean age at first injection (range), years	30 (16–45)	36 (29–46)	33 (16–46)
Male:Female	2:4	4:1	6:5
Clinical data
Ambulatory	0	0	0
Swallowing problems	2 (66.7%)	1 (33.3%)	3 (27.3%)
Noninvasive ventilation	3 (60.0%)	2 (40.0%)	5 (45.5%)
Scoliosis	6 (100%)	5 (100%)	11 (100%)
Spondylodesis	4 (66.7%)	2 (33.3%)	6 (54.5%)
Treatment goals (stabilization/ improvement of)
Hand strength	5 (62.5%)	3 (37.5%)	8 (72.7%)
Arm strength	2 (50.0%)	2 (50.0%)	4 (36.4%)
Respiratory function	4 (66.7%)	2 (33.3%)	6 (54.5%)
Swallowing	2 (100%)	0	2 (18.2%)
Sitting stability	0	1 (100%)	1 (9.1%)
Functional scales
HFMSE score, mean (± SD)
Baseline	0.2 (0.4), n = 6	3.2 (2.7), n = 5	1.5 (2.4), n = 11
2 months	0 (0), n = 4	2.5 (2.6), n = 4	1.3 (2.0), n = 8
6 months	0.3 (0.4), n = 4	0.0 (0.0), n = 2	0.2 (0.4), n = 6
10 months	0.5 (0.5), n = 2	n = 0	0.5 (0.5), n = 2
14 months	0.5 (0.5), n = 2	4.0 (0.0), n = 1	1.7 (1.7), n = 3
RULM score, mean (± SD)
Baseline	7.1 (6.2), n = 6	12.5 (12.1), n = 5	9.5 (9.7), n = 11
2 months	6.4 (6.2), n = 4	7.9 (7.7), n = 4	7.1 (7.0), n = 8
6 months	10.3 (6.2), n = 4	1.0 (1.0), n = 2	7.2 (6.7), n = 6
10 months	8.0 (8.0), n = 2	n = 0	8.0 (8.0), n = 2
14 months	8.0 (8.0), n = 2	7.0 (0.0), n = 1	7.7 (6.5), n = 3
ALS-FRS-R score, mean (± SD)
Baseline	22.0 (6.4), n = 6	24.0 (4.9), n = 5	22.9 (5.9), n = 11
2 months	19.8 (6.8), n = 4	24.0 (5.5), n = 4	21.9 (6.6), n = 8
6 months	20.5 (7.4), n = 4	19.5 (4.5), n = 2	20.2 (6.6), n = 6
10 months	18.0 (9.0), n = 2	n = 0	18.0 (9.0), n = 2
14 months	18.0 (9.0), n = 2	28.0 (0.0), n = 1	21.3 (8.7), n = 3

Percentages with respect to total number in subgroup. ALS-FRS-R, amyotrophic lateral sclerosis functional rating scale-revised; HFMSE, Hammersmith functional motor scale expanded; RULM, revised upper limb module.