Table 3.

Model parameters

		Fixed Effects				Random effects
	n	Intercept (SE)	95% CI Intercept	Slope	95% CI Slope	Std. dev. Intercept	Std. dev. Slope
FEV1
SMA type 1c	6	42.13 (4.58)	33.89; 50.59	−0.40	−1.42; 0.60 (n.s.)	6.11	1.12
SMA type 2a	46	61.71 (4.51)	52.44; 70.60	−1.29	− 1.78; − 0.81	24.35	1.21
SMA type 2b	34	81.37 (6.15)	68.36; 93.53	−1.37	−2.04; − 0.73	25.06	1.18
SMA type 3a	41	97.61 (6.30)	84.80; 110.02	−0.73	−1.11; − 0.35	23.20	0.64
SMA type 3b	32	100.35 (9.17)	81.91; 118.90	−0.11*	− 0.55; 0.32 *(n.s.)	16.62	n/a*
FVC
SMA type 1c	5	49.71 (7.34)	34.65; 68.07	−1.15	−3.29; 0.70 (n.s.)	12.60	1.70
SMA type 2a	47	64.20 (5.29)	53.65; 74.64	−1.32	−1.90; −0.76	28.46	1.39
SMA type 2b	34	84.53 (6.07)	71.85; 96.50	−1.40	−2.10; −0.71	23.68	1.28
SMA type 3a	43	96.65 (6.17)	84.08; 108.73	−0.67	−1.06; − 0.31	23.14	0.63
SMA type 3b	34	109.00 (7.42)	94.46; 123.50	−0.23*	−0.58; 0.11* (n.s.)	15.35	n/a*
VC
SMA type 1c	6	44.09 (7.06)	28.41; 60.01	−0.78	−2.35; 0.63 (n.s.)	12.84	1.21
SMA type 2a	32	61.01 (4.62)	51.78; 70.16	−1.57	−2.23; −0.94	23.17	1.40
SMA type 2b	22	85.54 (6.98)	69.33; 98.18	−1.65	−2.59; −0.60	25.04	1.46
SMA type 3a	16	96.34 (9.05)	78.17; 114.60	−1.06	−1.71; −0.45	30.07	0.98
SMA type 3b	4	80.99 (19.67)	35.90; 124.82	0.21	−0.77; 1.23 (n.s.)	35.12	0.75

Legend: Model parameter estimates, standard errors, and confidence intervals for the linear mixed-effects models are shown. n: number of patients in each group

SE standard error, CI confidence interval, Std. dev standard deviation, n.s. slope parameter is not significant; n/a not available

^* due to a too limited number of repeated-measurements ‘age at measurement’ was omitted as a random factor from the mixed-effects model. The slope parameter (i.e. the annual rate of decline in % of predicted) will therefore likely be an overestimation of the true value