Table 7 Frequency of ranking symptom in top 5 by disease severity of patient
From: Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale
| n (%) | Disease severity based on responses to patient/caregiver reported 5-domain NPCCSS in the survey |
Overall (n = 49) | ||
|---|---|---|---|---|
| ≤ 4 (n = 7) | 5–19 (n = 36) | ≥ 20 (n = 6) | ||
| Ambulation | 6 (85.7) | 31 (86.1) | 4 (66.7) | 41 (83.7) |
| Fine motor skills | 2 (28.6) | 17 (47.2) | 3 (50.0) | 22 (44.9) |
| Speech | 4 (57.1) | 26 (72.2) | 2 (33.3) | 32 (65.3) |
| Swallow | 3 (42.9) | 26 (72.2) | 6 (100.0) | 35 (71.4) |
| Cognition | 5 (71.4) | 16 (44.4) | 2 (33.3) | 23 (46.9) |