Table 1 Patient demographics and general characteristics per age-at-onset subgroup

Gender, n (%) female

3 (50)

7 (88)

28 (55)

25 (60)

10 (40)

Age, years:

At neurological onset

 n^a

–

8

50

40

18

 Mean (SD)

–

1.1 (0.7)

4.1 (1.2)

9.4 (2.6)

24.2 (8.8)

At diagnosis

 n^a

4^c

7^c

49

39

24

 Mean (SD)

0.1 (0.1)

1.3 (1.5)

4.6 (5.5)

11.5 (7.4)

29.3 (9.3)

At last follow up^b

 n^a

–

–

19

21

17

 Mean (SD)

–

–

11.6 (8.9)

20.2 (9.0)

39.5 (9.2)

At death

 n^a

6

8

30

19

8

 Mean (SD)

0.19 (0.22)

5.6 (2.0)

13.4 (6.7)

25.9 (8.9)

33.7 (6.2)

Number (%)^d with NP-C genetic information

1 (17)

8 (100)

41 (80)

36 (86)

16 (64)

Number (%)^d treated with miglustat

–

2 (25)

17 (33)

20 (48)

12 (48)