Table 5.

Preferences for SMA treatment by sub-groups

	SMA types			Age groups			Currently under gene therapy		Currently under Spinraza® (nusinersen) treatment
	Type I (n = 21)	Type II (n = 48)	Type III (n = 29)	≤ 2 years (n = 16)	3–17 years (n = 10)	≥ 18 years (n = 75)	Yes (n = 10)	No (n = 91)	Yes (n = 59)	No (n = 42)
Level of change in motor function (ref: stable)
Better	0.80*	0.41*	0.92*	0.68*	0.95*	0.61*	0.46	0.67*	0.79*	0.45*
Change in breathing function (ref: stable)
Better	1.16*	0.75*	0.58*	1.71*	− 0.12	0.80*	1.47*	0.75*	0.77*	0.78*
Drug is approved for: (ref: pediatric and adult patients with SMA)
Pediatric patients with SMA under 2 years of age	− 0.76*	− 1.51*	− 1.35*	− 0.10	− 0.28	− 1.68*	0.30	− 1.44*	− 1.33*	− 1.16*
Route and frequency of treatments (ref: intrathecal spinal injection—3–6 times per year)
Intravenous injection—one-time treatment	0.82*	0.42*	0.44*	1.69*	0.19	0.38*	2.03*	0.39*	0.39*	− 0.65*
Oral—daily medication	0.98*	0.82*	0.76*	1.01*	0.33	0.92*	0.65	0.87*	0.87*	0.05
Treatments potential risk (ref: mild risk)
Moderate risk	− 0.73*	− 0.81*	− 0.70*	− 0.79*	− 0.68	− 0.84*	− 0.34	− 0.84*	− 0.73*	− 0.81*
Serious, or life-threatening risk	− 1.91*	− 2.11*	− 1.43*	− 3.03*	− 1.55*	− 1.85*	− 2.83*	− 1.85*	− 1.61*	− 2.22*

^*p < 0.05; **p < 0.01; ***p < 0.001