Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review.

In juvenile patients, a clear trend towards reduced disease progression with miglustat treatment compared with the control group was observed, although this was not significant (increase in annual disability score [95% CI]: 0.34 [0.16–0.51] and 0.78 [0.50–1.06], respectively; Fig. 5 ).

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In patients treated for ≥1 year, linear regression modeling showed that miglustat treatment reduced disease progression in infantile patients and showed a trend towards reduced disease progression in juvenile patients in comparison with those who had no treatment or were treated for < 1 year.

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