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