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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