Table 1.

Demographics and baseline characteristics.

Variable	Level	Total (n = 13)
Age	Median (IQR) (min, max)	36.6 (33, 43) (18, 59)
Gender	Women	6 (46.2%)
	Men	7 (53.8%)
Baseline 6-min walk distance (meters)	Median (IQR) (min, max)	297 (203, 387) (107, 495)
Baseline SMA-FRS (normal range 0–40)	Median (IQR) (min, max)	31 (29, 34) (26, 36)
Baseline HFMSE (normal range 0–66)	Median (IQR) (min, max)	49 (45, 52) (23, 59)
SMN2 copies	3	4 (30.8%)
	4	8 (61.5%)
	5	1 (7.7%)