Table 1.

Baseline demographic and clinical characteristics (all enrolled patients)

	ERT experienced			ERT naïve
	Cohort 1	Cohort 2	Cohort 4	Cohort 3
	2–6 years prior ERT	non-ambulatory with ≥ 2 years prior ERT	≥ 7 years prior ERT	(n = 6)
	(n = 11)	(n = 6)	(n = 6)
Age, years
n	11	6	6	6
Mean (SD)	49.4 (9.5)	41.5 (18.1)	40.8 (17.0)	49.3 (15.1)
Median (range)	50.0 (28–66)	49.5 (18–57)	43.0 (20–65)	51.0 (24–65)
Sex, M:F	9:2	4:2	2:4	1:5
Race, n (%)
White	8 (72.7)	3 (50.0)	5 (83.3)	1 (16.7)
Missing	3 (27.3)	3 (50.0)	1 (16.7)	5 (83.3)
Time since Pompe diagnosis, years
n	11	6	6	6
Mean (SD)	7.7 (5.1)	15.9 (13.1)	13.0 (4.3)	5.2 (4.7)
Median (range)	6.3 (3–20)	12.5 (6–40)	12.8 (8–20)	3.7 (1–14)
Time on alglucosidase alfa, years
n	11	6	6	N/Aa
Mean (SD)	5.1 (1.3)	10.1 (4.8)	10.6 (2.1)	N/Aa
Median (range)	6.3 (3–20)	10.0 (5–16)	10.1 (8–14)	N/Aa
6MWD, % predicted
n	10	N/A	6	6
Mean (SD)	61.0 (13.43)	N/A	59.0 (21.44)	67.8 (12.61)
Median (range)	62.8 (35–83)	N/A	64.6 (17–80)	67.0 (54–89)
6MWD, m
n	10	N/A	6	6
Mean (SD)	397.2 (96.80)	N/A	387.3 (161.28)	396.0 (75.20)
Median (range)	394.5 (220–544)	N/A	431.0 (104–541)	395.2 (267–480)
Sitting FVC, % predicted
n	10	3	6	6
Mean (SD)	52.6 (13.9)	42.3 (28.2)	65.3 (21.1)	57.2 (20.8)
Median (range)	53.0 (31–70)	39.0 (16–72)	66.0 (31–88)	59.0 (31–79)
MMT lower extremity score
n	9	N/A	6	5
Mean (SD)	31.8 (1.9)	N/A	27.3 (3.7)	29.0 (1.7)
Median (range)	32.0 (30–34)	N/A	28.0 (21–30)	30.0 (26–30)

6MWD 6-min walking distance, CFBL change from baseline, ERT enzyme replacement therapy, FVC forced vital capacity, MMT manual muscle test, N/A not applicable, SD standard deviation

^aN = 1 ERT-naïve patient received one dose of alglucosidase alfa > 6 months prior to study entry