Table 2.

Longitudinal change of prospective outcomes following nusinersen treatment.

Measure	Time/comparison	N	Estimate (baseline/change from baseline)#	95% CI	p-value
Primary outcome
MVICT (Kg)	Baseline	13	6.75	4.3, 9.19
	2 months—baseline	13	0.36	−0.2, 0.92	0.2051
	6 months—baseline	13	0.29	−0.27, 0.85	0.3057
	10 months—baseline	13	0.52	−0.04, 1.08	0.0672
	14 months—baseline	9	0.2	−0.43, 0.84	0.5222
Functional scales
HFMSE	Baseline	13	45.85	40.41, 51.28
	2 months—baseline	13	2.77	0.84, 4.69	0.0058
	6 months—baseline	13	2.46	0.54, 4.39	0.0134
	10 months—baseline	13	2.08	0.15, 4.00	0.0351
	14 months—baseline	10	2.6	0.5, 4.69	0.0165
SMA-FRS	Baseline	13	31.23	29.47, 32.99
	2 months—baseline	13	1.69	0.42, 2.96	0.0102
	6 months—aseline	13	1.77	0.5, 3.04	0.0074
	10 months—baseline	13	1.23	−0.04, 2.50	0.0575
	14 months—baseline	11	1.57	0.23, 2.92	0.0224
Physical function
Six minute walk test (meters)	Baseline	11	262.88	171.42, 354.35
	2 months—baseline	13	19.49	2.91, 36.07	0.0223
	6 months—baseline	13	25.27	8.69, 41.85	0.0037
	10 months—baseline	13	19.19	2.61, 35.77	0.0243
	14 months—baseline	10	15.88	−2.16, 33.92	0.0829
Forced vital capacity (liters)	Baseline	13	4.07	3.38, 4.77
	2 months—baseline	13	0.07	−0.04, 0.18	0.2206
	6 months—baseline	13	0.16	0.04, 0.27	0.0081
	10 months—baseline	13	0.12	0.01, 0.24	0.0332
	14 months—baseline	10	0.17	0.05, 0.3	0.0072
Electrophysiological measures of motor unit connectivity
Compound muscle action potential amplitude (millivolts)	Baseline	13	8.42	7.16, 9.68
	2 months—baseline	13	0.08	−0.22, 0.39	0.5766
	6 months—baseline	13	0.35	0.05, 0.66	0.0231
	10 months—baseline	13	0.41	0.10, 0.71	0.0095
	14 months—baseline	10	0.47	0.14, 0.80	0.0060
Average single motor unit potential amplitude (microvolts)	Baseline	11	80.83	71.14, 90.51
	2 months—baseline	13	5.13	0.35, 9.91	0.0359
	6 months—baseline	13	3.76	−1.02, 8.55	0.1196
	10 months—baseline	13	6.69	1.91, 11.47	0.0072
	14 months—baseline	10	8.93	3.73, 14.13	0.0012
Motor unit number estimation	Baseline	11	106.17	88.14, 124.21
	2 months—baseline	13	−2.17	−7.83, 3.48	0.4419
	6 months—baseline	13	−1.64	−7.29, 4.01	0.5623
	10 months—baseline	13	−2.48	−8.13, 3.17	0.3806
	14 months—baseline	10	−5.22	−11.37, 0.93	0.0940

^#Positive values indicate increase compared to baseline, and negative values indicate decrease from baseline at each time point (2, 6, 10, and 14 months).