Table 1.

Assessment methods.

Assessment method	Events (N)	Participants per event
Neurobehavioral symptoms
Caregiver focus groups	3	Event 1 N = 3
		Event 2 N = 2
		Event 3 N = 4
Videos of affected children	2	Event 1 N = 3
		Event 2 N = 2*
Individual caregiver interviews	3	1
Patient advocacy group meeting	1	3 patient advocacy groups
		Group 1 N = 3
		Group 2 N = 1
		Group 3 N = 1
Expert consultation	5	1
Medical chart review	N/A	12
Parent advisory board	1	N = 7
Family Impact
Caregiver focus groups	3	Event 1 N = 3
		Event 2 N = 2
		Event 3 N = 4
Individual caregiver interviews	3	1
Patient advocacy group meeting	1	3 patient advocacy groups
		Group 1 N = 3
		Group 2 N = 1
		Group 3 N = 1
Expert consultation	5	1
Parent advisory board	1	N = 7