Table 1.

Demographics, genotype and phenotype characteristics, and baseline functional assessment.

Variable	Level	Total (n = 19)
Age	Mean (Std) (min, max)	39.7 (13.9) (21.3, 64.8)
Sex	Women	12 (63.2%)
	Men	7 (36.8%)
SMN2 copies	2	1 (5.3%)
	3	16 (84.2%)
	4	2 (10.5%)
Age of onset (months)	Mean (Std) (min, max)	27 (34) (2–156)
SMA type	2	9 (47%)
	3	10 (53%)
Age at loss of ambulation (SMA 3) (years)	Mean (Std) (min, max)	25.8 (18.3) (8, 57)
Baseline SMA-FRS	Mean (Std) (min, max)	11.2 (10) (0, 30)
Baseline hammersmith	Mean (Std) (min, max)	3.5 (5.7) (0, 18)
Baseline RULM	Mean (Std) (min, max)	12.4 (11.5) (0, 37)