Table 1.

Demographics and baseline characteristics (safety set)

	<2 years (N = 2)	2 to <12 years (N = 5)	12 to <18 years (N = 5)	≥18 years (N = 6)	All (N = 18)
Age (years)
Mean (SD)	1.5 (0.2)	7.7 (2.4)	13.9 (1.2)	21.1 (2.3)	13.2 (7.1)
Min, max	1.3, 1.6	5.0, 11.0	13.0, 15.4	19.0, 24.0	1.3, 24.0
Sex
Male	2 (100.0%)	4 (80.0%)	2 (40.0%)	1 (16.7%)	9 (50.0%)
Female	0	1 (20.0%)	3 (60.0%)	5 (83.3%)	9 (50.0%)
Age at HT-1 diagnosis (months)
Mean (SD)	8.0 (2.1)	8.2 (6.4)	7.0 (4.1)	8.8 (5.3)	8.1 (4.8)
Age at nitisinone treatment start (months)
Mean (SD)	8.5 (2.1)	8.2 (6.4)	7.2 (4.3)	15.3 (14.3)	10.3 (9.4)

SD standard deviation