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Fig. 3 | Orphanet Journal of Rare Diseases

Fig. 3

From: Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect

Fig. 3

Changes in total NP-C disability score for each patient from baseline (diagnosis) to last follow up. Each point represents change in total disability score in individual patients according to delay between diagnosis and last follow up. A positive change in disability score indicates clinical worsening. Patients who discontinued miglustat after < 2 years due to neurological worsening were excluded (n = 4). For three patients (2, 6, and 16), change in disability score was measured between age at miglustat onset and age at last examination, as delay between diagnosis and miglustat onset exceeded 1 year. The period between diagnosis and last follow up and the duration of miglustat treatment were associated with change in disability score from baseline (p < 0.001 for both variables). Clinical score at diagnosis and age at neurological onset did not show any statistically significant relationship (p = 0.34 and 0.30, respectively)

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