. Author manuscript; available in PMC: 2020 Aug 26.

Published in final edited form as: CNS Drugs. 2019 Jul;33(7):677–683. doi: 10.1007/s40263-019-00642-2

Table 1.

Participant information (N = 14)

	Mean ± Standard Deviation	Range

Age at diagnosis (years)	9.07 ± 5.65	2 − 20
Age at baseline (years)	15.07 ± 5.53	4 − 23
Niemann-Pick Type C1 Total Severity	19.29 ± 7.46	5 − 32
Score at Baseline
	n (%)
Female	7 (50%)
White	14 (100%)
Non-Latino/Non-Hispanic	13 (93%)
Hearing loss at baseline	11 (79%)
Seizures	5 (36%)
Miglustat	11 (79%)