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TITLE: Clinical experience with miglustat therapy in pediatric patients with Niemann Pick disease type C: A case series  Full Text
AUTHORS: Pineda, M; Perez Poyato, MS; O'Callaghan, M; Vilaseca, MA; Pocovi, M; Domingo, R; Ruiz Portal, LR; Verdu Perez, AV; Temudo, T; Gaspar, A; Garcia Penas, JJG; Roldan, S; Martin Fumero, LM; Blanco de la Barca, OB; Garcia Silva, MTG; Macias Vidal, J; Coll, MJ;
PUBLISHED: 2010, SOURCE: MOLECULAR GENETICS AND METABOLISM, VOLUME: 99, ISSUE: 4
INDEXED IN: Scopus WOS CrossRef