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Displaying 3 results.
    • Journal article
    Phase I/II Trial of Liver Derived Mesenchymal Stem Cells in Pediatric Liver Based Metabolic Disorders: A Prospective, Open Label, Multicenter, Partially Randomized, Safety Study of One Cycle of Heterologous Human Adult Liver-Derived Progenitor Cells (HepaStem®) in Urea Cycle Disorders and Crigler-Najjar Syndrome patients.
    Smets, Françoise[UCL] Dobbelaere, Dries McKiernan, Patrick Dionisi-Vici, Carlo Broué, Pierre Jacquemin, Emmanuel Lopes, Ana Isabel Gonçalves, Isabel Mandel, Hanna Pawlowska, Joanna Kamińska, Diana Shteyer, Eyal Torre, Giuliano Shapiro, Riki Eyskens, François Clapuyt, Philippe[UCL] Gissen, Paul Pariente, Danièle Grunewald, Stephanie Yudkoff, Marc Binda, Maria Mercedes Najimi, Mustapha[UCL] Belmonte, Nathalie Vos, Beatrice De Thonnard, Joelle Sokal, Etienne[UCL] (2019) Transplantation — Vol. 103, no. 9, p. 1903-1915 (2019)
    • Journal article
    Phenotype, treatment practice and outcome in the cobalamin-dependent remethylation disorders and MTHFR deficiency: Data from the E-HOD registry.
    Huemer, Martina Diodato, Daria Martinelli, Diego Olivieri, Giorgia Blom, Henk Gleich, Florian Kölker, Stefan Kožich, Viktor Morris, Andrew A Seifert, Burkhardt Froese, D Sean Baumgartner, Matthias R Dionisi-Vici, Carlo EHOD consortium Martin, Carlos Alcalde Baethmann, Martina Ballhausen, Diana Blasco-Alonso, Javier Boy, Nikolas Bueno, Maria Burgos Peláez, Rosa Cerone, Roberto Chabrol, Brigitte Chapman, Kimberly A Couce, Maria Luz Crushell, Ellen Dalmau Serra, Jaime Diogo, Luisa Ficicioglu, Can García Jimenez, Maria Concepcion García Silva, Maria Teresa Gaspar, Ana Maria Gautschi, Matthias González-Lamuño, Domingo Gouveia, Sofia Grünewald, Stephanie Hendriksz, Chris Janssen, Mirian C H Jesina, Pavel Koch, Johannes Konstantopoulou, Vassiliki Lavigne, Christian Lund, Allan M Martins, Esmeralda G Meavilla Olivas, Silvia Mention, Karine Mochel, Fanny Mundy, Helen Murphy, Elaine Paquay, Stéphanie[UCL] Pedrón-Giner, Consuelo Ruiz Gómez, Maria Angeles Santra, Saikat Schiff, Manuel Schwartz, Ida Vanessa Scholl-Bürgi, Sabine Servais, Aude Skouma, Anastasia Tran, Christel Vives Piñera, Inmaculada Walter, John Weisfeld-Adams, James (2019) Journal of Inherited Metabolic Disease — Vol. 42, no.2, p. 333-352 (2019)
    • Journal article
    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 Seminara, Jennifer Summar, Marshall Hoffmann, Georg F. Kölker, Stefan Burgard, Peter Sokal, Etienne[UCL] (2019) Journal of Inherited Metabolic Disease — Vol. 42, no. 1, p. 93-106 (2019)