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Disease duration and survival in Brazilian Niemann-Pick disease type C patients: Preliminary data on potential impact of miglustatGiugliani, Luciana ; Van der Linder, Vanesa ; Van Der Linden, Helio ; Lourenço, Charles Marques ; de Araújo Leão, Emília Katiane Embiruçu ; Arita, Juliana Harumi ; Doriqui, Maria Julia Rodovalho ; Neto, Pedro Braga ; de Souza, Carolina Fischinger Moura ; Horovitz, Dafne Dain Gandelman ; Santos, Mara Lucia ; Grillo, Eugenio ; da Silva, Raquel Tavares Boy ; de Oliveira Abrão, Janaina Bianca ; Trapp, Franciele Barbosa ; Giugliani, RobertoMolecular genetics and metabolism, 2016-02, Vol.117 (2), p.S50-S51 [Periódico revisado por pares]Elsevier IncTexto completo disponível |