Valuation of treatments for rare diseases: a systematic literature review of societal preference studies by Dabbous, O, Chachoua, L, Aballéa, S, Sivignon, M, Persson, U, Petrou, S, Richardson, J, Simoens, S, Toumi, M

Health economic evaluation of gene replacement therapies: methodological issues and recommendations by Aballéa, S, Thokagevistk, K, Velikanova, R, Simoens, S, Annemans, L, Antonanzas, F, Auquier, P, François, C, Fricke, F-U, Malone, D, Millier, A, Persson, U, Petrou, S, Dabbous, O, Postma, M, Toumi, M

RESTORE: a prospective multinational registry of patients with genetically confirmed spinal muscular atrophy - rationale and study design by Finkel, RS, Day, JW, De Vivo, DC, Kirschner, J, Mercuri, E, Muntoni, F, Shieh, PB, Tizzano, E, Desguerre, I, Quijano-Roy, S, Saito, K, Droege, M, Dabbous, O, Khan, F, Renault, L, Anderson, FA, Servais, L