Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C
Bénédicte Héron*, Vassili Valayannopoulos, Julien Baruteau, Brigitte Chabrol, Hélène Ogier, Philippe Latour, Dries Dobbelaere, Didier Eyer, François Labarthe, Hélène Maurey, Jean-Marie Cuisset, Thierry B de Villemeur, Frédéric Sedel and Marie T Vanier