Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study

Richard S. Finkel, Claudia A. Chiriboga, Jiri Vajsar, John W Day, Jacqueline Montes, Darryl C. De Vivo, Kathie M. Bishop, Richard Foster, Yingying Liu, Daniela Ramirez‐Schrempp, Eugene Schneider, C. Frank Bennett, Janice Wong, Wildon Farwell