Figure 2
From: Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C
Changes over time in individual patient composite scores on the NP-C disability scale during miglustat treatment. Patients with a) early-infantile, b) late-infantile and c) juvenile-onset disease; data included for NPC1 patients only; no disability scale data available from early-infantile patient 5, who died aged 2 years and 9 months; *patient 12 died at 13 years 11 months of age.