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Fig. 4 | Orphanet Journal of Rare Diseases

Fig. 4

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

Fig. 4

The inclusion criteria for three clinical trials were modeled on plots of age versus total severity score (minus hearing). a Miglustat trial, ages > 12; b Arimoclomol trial ages 2–18 and c 2-Hydroxy-beta-cyclodextrin ages 4–21. The plots depict the study cohort with these different inclusion criteria indicated with the dotted lines. Included patients are in black, excluded patients in white

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172