Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study - Finkel, RS; Chiriboga, CA; Vajsar, J; Day, JW; Montes, J; De Vivo, DC; Yamashita, M; Rigo, F; Hung, G; Schneider, E; Norris, DA; Xia, S; Bennett, CF; Bishop, KM (Lancet, 2016)