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Table 3 Ranking of clinical subdomains most important to parents of NPC1 patients and expert clinicians. The top 5 subdomains selected by each group completing the survey are shaded in blue

From: Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172