Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders-A successful strategy for clinical research of rare diseases.
Posset R, Garbade SF, Boy N, Burlina AB, Dionisi-Vici C, Dobbelaere D, Garcia-Cazorla A, de Lonlay P, Teles EL, Vara R, Mew NA, Batshaw ML, Baumgartner MR, McCandless SE, Seminara J, Summar M, Hoffmann GF, Kölker S, Burgard P; Additional individual contributors of the UCDC and the E-IMD consortium.
Posset R, et al. Among authors: burlina ab.
J Inherit Metab Dis. 2019 Jan;42(1):93-106. doi: 10.1002/jimd.12031.
J Inherit Metab Dis. 2019.
PMID: 30740724
Free PMC article.