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Effects of Miglustat Therapy on Neurological Disorder and Survival in Early-Infantile Niemann-Pick Disease Type C: A National French Retrospective Study

Cécile Freihuber, Bahia Dahmani-Rabehi,Anaïs Brassier,Pierre Broué,Claude Cances,Brigitte Chabrol,Didier Eyer,François Labarthe,Philippe Latour,Thierry Levade,Samia Pichard,Caroline Sevin,Marie T. Vanier,Bénédicte Héron

ORPHANET JOURNAL OF RARE DISEASES（2023）

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Niemann-Pick Disease
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