Table 1.

Demographic characteristics of human subjects.

Cohort	Sample type	Patient type	GM1 gangliosidosis	Control	p value
Evaluation of ganglioside GM1	Plasma	Age (range, median; mean ± SD)	3–20, 11; 11 ± 6.2	1–18, 10; 9 ± 5.6	0.3130a
		Male/Female (n/n)	5/5	20/10	0.5716b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
	CSF	Age (range, median)	3–20, 11; 11 ± 6.2	<1–20, 6; 8 ± 6.4	0.151c
		Male/Female (n/n)	5/5	9/11	1b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
Discovery of oligosaccharide biomarkers	Urine	Age (range, median)	3–20, 11; 11 ± 6.2	1–17, 13; 12 ± 4.9	0.7178c
		Male/Female (n/n)	5/5	5/5	1b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
	Plasma	Age (range, median)	3–20, 11; 11 ± 6.2	1–18, 11; 10 ± 6.3	0.7038a
		Male/Female (n/n)	5/5	5/5	1b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
	CSF	Age (range, median)	3–20, 11; 11 ± 6.2	<1–20, 6; 8 ± 6.4	0.151a
		Male/Female (n/n)	5/5	9/11	1b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
Establishment of H3N2b reference range	Urine	Age (range, median)	3–20, 11; 11 ± 6.2	<1–26, 10; 10 ± 7.0	0.4615a
		Male/Female (n/n)	5/5	19/44	0.3797b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
Establishment of H3N2b reference range	Plasma	Age (range, median)	3–20, 11; 11 ± 6.2	<1–23, 9; 9 ± 5.9	0.3095a
		Male/Female (n/n)	5/5	21/29	0.9072b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
Establishment of H3N2b reference range	CSF	Age (range, median)	3–20, 11; 11 ± 6.2	<1–19, 8; 9 ± 6.6	0.225a
		Male/Female (n/n)	5/5	18/14	1b
		Late infantile (n)	4		–
		Juvenile (n)	6		–
Single patient in expanded access	Urine, plasma, CSF	Age	10		–
		Gender	Female		–
		GM1 gangliosidosis type	Juvenile		–
		Dose	1.5 × 1013 vg/kg		–

^aMann–Whitney U-test.

^bChi-square test.

^cTwo sample t-test.