Table 4.

Longitudinal change of outcomes.

Measure	Time/Comparison	N	Estimate (baseline/change from baseline)#	95% CI	p-value
Pulmonary function
Actual FVC	Baseline	19	1.94	1.3, 2.58
	2 months–baseline	17	−0.02	−0.11, 0.08	0.7296
	6 months–baseline	19	−0.02	−0.11, 0.07	0.6628
	10 months–baseline	17	−0.02	−0.11, 0.07	0.6762
	14 months–baseline	12	0.02	−0.09, 0.12	0.7733
NIF	Baseline	19	−37.84	−46.79, −28.89
	2 months–baseline	17	−7.46	−17.88, 2.96	0.1571
	6 months–baseline	18	−5.74	−15.97, 4.49	0.2658
	10 months–baseline	17	−5.06	−15.47, 5.34	0.3343
	14 months–baseline	12	−9.14	−20.83, 2.55	0.1232
Functional scales
Hammersmith	Baseline	19	3.47	0.01, 6.93
	2 months–baseline	18	0.77	−0.29, 1.83	0.1515
	6 months–baseline	19	0.74	−0.3, 1.78	0.1624
	10 months–baseline	19	0.32	−0.73, 1.36	0.5468
	14 months–baseline	12	0.11	−1.11, 1.32	0.8606
SMA-FRS	Baseline	19	11.16	6.61, 15.7
	2 months–baseline	18	−0.26	−1.28, 0.77	0.6510
	6 months–baseline	19	−0.58	−1.59, 0.43	0.2551
	10 months–baseline	19	−0.58	−1.59, 0.43	0.2551
	14 months–baseline	14	−0.98	−2.1, 0.13	0.1306
RULM	Baseline	19	12.42	6.91, 17.93
	2 months–baseline	18	1.31	0.24, 2.39	0.0171
	6 months–baseline	19	0.89	−0.16, 1.95	0.0946
	10 months–baseline	19	0.95	−0.1, 2	0.0771
	14 months–baseline	12	0.27	−0.96, 1.5	0.6637
Strength measurement
Key pinch	Baseline	17	0.55	0.05, 1.05
	2 months–baseline	18	0.18	0.07, 0.29	0.0019
	6 months–baseline	19	0.1	−0.01, 0.21	0.0748
	10 months–baseline	19	0.1	−0.005, 0.21	0.0617
	14 months–baseline	12	0.01	−0.11, 0.14	0.8484
Handgrip	Baseline	17	1.31	0.39,2.23
	2 months–baseline	18	0.2	−0.21,0.61	0.3405
	6 months–baseline	19	0.43	0.03, 0.84	0.0377
	10 months–baseline	19	0.13	−0.28, 0.54	0.5236
	14 months–baseline	12	0.11	−0.36, 0.57	0.6514
Electrophysiological measures of motor unit connectivity
CMAP	Baseline	19	2.54	1.34, 3.74
	2 months–baseline	18	0.12	−0.14, 0.38	0.3446
	6 months–baseline	19	0.22	−0.04, 0.47	0.0949
	10 months–baseline	19	0.29	0.04, 0.55	0.0238
	14 months–baseline	13	0.32	0.03, 0.61	0.0308
For the measurements below (SMUP and MUNE), only 13 patients were included in the analysis
SMUP	Baseline	12	98.49	72.52, 124.46
	2 months–baseline	11	2.69	−1.35, 6.72	0.1852
	6 months–baseline	12	3.31	−0.6, 7.23	0.0949
	10 months–baseline	12	5.09	1.18, 9.01	0.0122
	14 months–baseline	6	6.98	1.99, 11.98	0.0074
MUNE	Baseline	12	40	19.44, 60.56
	2 months–baseline	11	−1.25	−4.13, 1.62	0.3816
	6 months–baseline	12	−1.17	−3.95, 1.62	0.4020
	10 months–baseline	12	0.08	−2.7, 2.87	0.9520
	14 months–baseline	6	−1.25	−4.81, 2.31	0.4807

Statistical method: linear mixed models were used to explore the change of these measures across time with random intercepts for each participant. Difference between each time point with baseline and 95 % CI were reported. #Positive values indicate increase compared to baseline, and negative values indicate decrease from baseline at each time point (2, 6, 10, and 14 months).