title: [Niemann-Pick type C disease: an early diagnosis for a therapeutic hope].
authors.affiliation: Centre de Référence des Maladies Métaboliques, Hôpital Robert Debré, 48 Boulevard Sérurier, 75019 Paris, France. helene.ogier@rdb.aphp.fr
authors.forename: H
authors.initials: H
authors.lastname: Ogier de Baulny
mesh_terms: D042241:Early Diagnosis; D015316:Genetic Therapy; D006801:Humans; D052556:Niemann-Pick Disease, Type C / Q000175:diagnosis* / Q000628:therapy; D047392:beta-Cyclodextrins
chemical_list: D047392:beta-Cyclodextrins