Table 8 Level of worsening considered meaningful across 5-domain NPCCSS
From: Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale
1-category meaningful, n (%) | 2-categories meaningful (if 1-category not meaningful), n (%) | |||
|---|---|---|---|---|
Yes | No | Yes | No | |
Ambulation (n = 26) | 23 (88.5) | 3 (11.5) | 3 (100) | 0 |
Fine Motor Skills (n = 25) | 22 (88.0) | 3 (12.0) | 3 (100) | 0 |
Speech (n = 26) | 21 (80.8) | 5 (19.2) | 4 (100) | 0 |
Swallow (n = 23) | 17 (73.9) | 6 (26.1) | 5 (83.3) | 1 (16.7) |
Cognition (n = 21) | 13 (61.9) | 8 (38.1) | 5 (100) | 0 |