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 ; Ah Mew, Nicholas ; Batshaw, Mark L ; Baumgartner, Matthias R ; McCandless, Shawn ; Seminara, Jennifer ; Summar, Marshall ; Hoffmann, Georg F ; Kölker, Stefan ; Burgard, Peter

Journal of inherited metabolic disease, 2018-07, p.1-13 [Periódico revisado por pares]

United States: Blackwell Publishing Ltd

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