Skip to main content

Table 2 Clinical characteristics of the NPC1 cohort

From: Identification of cerebral spinal fluid protein biomarkers in Niemann-Pick disease, type C1

 

n

Mean

p-value*

Median (range)

NPC NSS (points)

28

16.8 ± 11.0

-

18.0 (0–42)

No Miglustat

14

20.0 ± 8.2

 

20.0 (0–32)

Miglustat

14

13.6 ± 12.8

0.07

13.6 (0–42)

ASIS (points/year)

26**

1.95 ± 2.26

-

1.07 (0.31–9.37)

No Miglustat

13

2.25 ± 2.69

 

1.31 (0.31–9.37)

Miglustat

13

1.64 ± 1.80

0.61

0.89 (0.31–6.09)

Age of Neurological Onset (years)

28

8.47 ± 12.02

-

3.5 (1.3–43)

No Miglustat

14

12.07 ± 15.79

 

5.0 (1.5–43)

Miglustat

14

4.86 ± 4.79

0.23

2.0 (1.3–16)

  1. *Mann–Whitney t-test
  2. **2 individuals with NPC NSS = 0