Table 1.

Neuropsychological and disease severity descriptive statistics for CLN3 cohort.

		All			Homozygous 1-kb deletion			Other genotypesa
		N	Range	Mean (SD) or Median [IQR]	N	Range	Mean (SD) or Median [IQR]	N	Range	Mean (SD) or Median [IQR]
Baseline
Vineland-3	ABC	22	20-110	61.9 (26.8)	10	26-82	55.1 (21.2)	10	20-88	59.4 (26.1)
	Comm	22	20-113	59.6 (30.0)	10	20-87	51.4 (25.2)	10	20-96	58.5 (30.2)
	DLS	22	20-106	57.4 (29.9)	10	20-85	49.8 (28.6)	10	20-85	55.5 (26.6)
	Social	22	20-120	70.6 (26.7)	10	36-92	65 (18.8)	10	20-102	68 (29.9)
VIQ		19	45-108	78.6 (20.2)	9	55-100	75 (17.6)	8	45-106	76.9 (22.7)
UBDRS	Physical	22	1-61	9.5 [4;21.5]	10	3-33	10.4 [6.8;19.5]	10	2-61	12 [4;37]
	Capability	22	1-14	6.5 [4.3;10]	10	4-11	6 [4.5;9]	10	1-14	6.5 [4.3;9.5]
	CGI	22	9.3-24.5	15.2 [12.0;19.8]	10	9.3-21	17.5 [12.8;19.5]	10	9.3-24.5	15.2 [12.0;21.6]
1-year Follow-up
Vineland	ABC	17	24-117	64 (26.5)	8	33-84	56.6 (20.2)	7	24-76	58.9 (22.9)
	Comm	17	20-109	58.9 (27.4)	8	20-83	51.6 (22.0)	7	20-79	53.9 (24.5)
	DLS	17	20-120	62.3 (29.4)	8	20-89	52.4 (28.2)	7	31-79	59.1 (18.9)
	Social	17	20-110	71.4 (27.1)	8	34-94	66.9 (22.05)	7	20-90	65.7 (29.2)
VIQ		8	72-103	89.6 (11.9)	3	72-92	80 (10.6)	3	81-103	93 (11.1)
UBDRS	Physical	13	1-36	7 [6;13]	7	3-36	9 [5.5;28.5]	5	6-13	7 [6;8]
	Capability	14	4-14	7 [6;7.8]	7	4-8	7 [5.5;7]	5	5-9	7 [6;7]
	CGI	14	11-22	15.5 [14;16.8]	7	15-22	16 [15.5;18.5]	5	12-17	14 [14;16]

^aExcluding 2 participants with vision-only phenotype.

ABC: adaptive behavior composite. CGI: clinical global impression. Comm: communication. DLS: daily living skills. IQR: interquartile range. SD: standard deviation. UBDRS: Unified Batten Disease Rating Scale. VIQ: verbal intelligence quotient.