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Table 2 Demographics and characteristics of patients enrolled in the NPC Registry (N = 472)

From: Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry

 

Patient characteristics of overall population

(N = 472)

Patients with age at neurological onset data

Early-infantile (< 2 years)

Late-infantile (2 to < 6 years)

Juvenile (6 to < 15 years)

Adolescent/adult onset (≥ 15 years)

Male: female, n (%a)

245 (51.9): 227 (48.1)

Age at enrollment

n

470

 Mean (SD), years

21.2 (15.0)

 Median (range), years

19.0 (0.2–71.8)

Age at onset of neurological symptoms

n (%a)

422 (100.0)

57 (13.5)

108 (25.6)

134 (31.8)

123 (29.1)

 Mean (SD), years

12.3 (11.8)

0.8 (0.7)

4.0 (1.2)

10.1 (2.6)

27.2 (11.0)

 Median (range), years

8.8 (0.0–71.8)

0.8 (0.0–2.0)

4.0 (2.0–6.0)

10.0 (6.0–15.0)

25.0 (15.0–71.8)

Age at diagnosis

n (%a)

292 (100.0)

37 (12.7)

84 (28.8)

87 (29.8)

84 (28.8)

 Mean (SD), years

3.3 (4.6)

8.3 (7.4)

16.3 (8.6)

33.5 (12.4)

 Median (range), years

1.6 (0.1–21.4)

6.7 (0.1–33.1)

13.9 (2.9–56.3)

31.8 (14.4–69.8)

  1. SD Standard deviation
  2. aPercentage based on patients with data (excluding missing responses)