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

Nusinersen versus sham control in later-onset spinal muscular atrophy by Mercuri, E, Darras, BT, Chiriboga, CA, Day, JW, Campbell, C, Connolly, AM, Iannaccone, ST, Kirschner, J, Kuntz, NL, Saito, K, Shieh, PB, Tulinius, M, Mazzone, ES, Montes, J, Bishop, KM, Yang, Q, Foster, R, Gheuens, S, Bennett, CF, Farwell, W, Schneider, E, De Vivo, DC, Finkel, RS