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Table 4 Mortality in untreated and miglustat-treated NP-C patients included in survival analysis by age at neurological disease onset

From: Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat

 

Death, n (%)

  

Untreated

 

Treated

 

n

(N = 97)

n

(N = 90)

Overall

 

74 (76)

 

3 (3)

Age category*

    

<2 years

27

27 (100)

4

0 (0)

2–11 years

48

40 (83)

37

2 (5)

>11 years

22

7 (32)

49

1 (2)

  1. *Age at neurological symptom onset in untreated patients, and age at treatment start in treated patients.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172