Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders—A successful strategy for clinical research of rare diseases

Posset, Roland ; Garbade, Sven F. ; Boy, Nikolas ; Burlina, Alberto B. ; Dionisi‐Vici, Carlo ; Dobbelaere, Dries ; Garcia‐Cazorla, Angeles ; de Lonlay, Pascale ; Teles, Elisa Leão ; Vara, Roshni ; Mew, Nicholas Ah ; Batshaw, Mark L. ; Baumgartner, Matthias R. ; McCandless, Shawn E. ; Seminara, Jennifer ; Summar, Marshall ; Hoffmann, Georg F. ; Kölker, Stefan ; Burgard, Peter ; Berry, Susan A. ; Burrage, Lindsay ; Coughlin, Curtis ; Diaz, George A. ; Gallagher, Renata C. ; Gropman, Andrea ; Harding, Cary O. ; Lee, Brendan ; Le Mons, Cynthia ; Lawrence Merritt, J. ; Nagamani, Sandesh C. S. ; Schulze, Andreas ; Stricker, Tamar ; Tuchman, Mendel ; Waisbren, Susan ; WeisfeldAdams, James ; Wong, Derek ; Yudkoff, Marc ; Arnoux, JeanBaptiste ; Bari, Ivo ; Bosch, Annet M. ; Chabrol, Brigitte ; Chakrapani, Anupam ; CortèsSaladefont, Elisenda ; Couce, Maria L. ; Eyskens, Francois ; Laet, Corine ; Meirleir, Linda ; Freisinger, Peter ; Gleich, Florian ; Grünewald, Stephanie ; Häberle, Johannes ; Hwu, WuhLiang ; Jalan, Anil ; Karall, Daniela ; Lindner, Martin ; Lund, Allan M. ; Martinelli, Diego ; Murphy, Elaine ; Mühlhausen, Chris ; Olivieri, Giorgia ; Ottolenghi, Chris ; Rodrigues, Esmeralda ; Rubert, Laura ; Sarajlija, Adrijan ; Schiff, Manuel ; Sokal, Etienne ; SykutCegielska, Jolanta ; Walter, John H. ; Williams, Monique ; Zeman, Jiri

Journal of inherited metabolic disease, 2019-01, Vol.42 (1), p.93-106 [Periódico revisado por pares]

Hoboken, USA: John Wiley & Sons, Inc

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