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TITLE: Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders—a successful strategy for clinical research of rare diseases
AUTHORS: Posset, R; Garbade, SF; Boy, N; Burlina, AB; Dionisi Vici, C; Dobbelaere, D; Garcia Cazorla, A; de Lonlay, P; Teles, EL; Vara, R; Ah Mew, N; Batshaw, ML; Baumgartner, MR; McCandless, S; Seminara, J; Summar, M; Hoffmann, GF; Kölker, S; Burgard, P; Berry, SA; Burrage, L; Coughlin, C; Diaz, GA; Gallagher, RC; Gropman, A; Harding, CO; Lee, B; Le Mons, C; Merritt, JL; Nagamani, SCS; Schulze, A; Stricker, T; Tuchman, M; Waisbren, S; Weisfeld Adams, J; Wong, D; Yudkoff, M; Arnoux, JB; Barić, I; Bosch, AM; Chabrol, B; Chakrapani, A; Cortès Saladefont, E; Couce, ML; de Laet, C; de Meirleir, L; Eyskens, F; Freisinger, P; Gleich, F; Grünewald, S; Häberle, J; Hwu, WL; Jalan, A; Karall, D; Lindner, M; Lund, AM; Martinelli, D; Murphy, E; Mühlhausen, C; Olivieri, G; Ottolenghi, C; Rodrigues, E; Rubert, L; Sarajlija, A; Schiff, M; Sokal, E; Sykut Cegielska, J; Walter, JH; Williams, M; Zeman, J; ...More
PUBLISHED: 2018, SOURCE: Journal of Inherited Metabolic Disease
INDEXED IN: Scopus