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