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Fig. 3 | Orphanet Journal of Rare Diseases

Fig. 3

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

Fig. 3

5-domain NPCCSS scores at Visit 1 and Visit 2. a Mean (SD) overall scores at Visit 1 and Visit 2 for all participants.* b Change in overall scores from Visit 1 to Visit 2 by length of follow-up time. c Mean (SD) individual scores for the five key sub-domains at Visit 1 and Visit 2, with mean change. NPCCSS: Niemann–Pick disease type C Clinical Severity Scale; SD: standard deviation. *One participant who withdrew early had relocated, but had an end-of-trial visit and is included in efficacy assessments. According to the statistical analysis plan, the NPC-001 study ended at a study site once the interventional NPC-002 study commenced at that specific site. The NPC-002 study had not started at the site of this particular participant when they relocated, therefore the end of study visit was planned (prior to completion of the study). The participant had a planned withdrawal visit after 162 days. Of the study withdrawals, only this participant had an end of study visit. Their screening visit and end of study visit are therefore included as per statistical analysis plan

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