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Table 7 Mean 5-domain NPCCSS score by participant videos

From: Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study

Participants*

MEAN 5-domain NPCCSS score (± SD)

Initial rating

Repeat rating

Participant A

5.85 (2.115)

7.00 (1.633)

Participant B

11.08 (2.326)

10.46 (1.713)

Participant C

18.15 (1.772)

18.85 (1.068)

Participant D

4.46 (1.391)

4.77 (2.204)

p value

 < 0.0001

 < 0.0001

  1. * Facets measures were as follows: participant C = 3.30 (most severe); participant B = 0.22; participant A = –1.32; participant D = –2.20 (least severe)
  2. NPCCSS: Niemann–Pick type C disease Clinical Severity Scale
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172