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

著者: Richard S. Finkel, Claudia A. Chiriboga, Jiri Vajsar, John W Day, Jacqueline Montes, Darryl C. De Vivo, Mason Yamashita, Frank Rigo, Gene Hung, Eugene Schneider, Daniel A Norris, Shuting Xia, C. Frank Bennett, Kathie M. Bishop - The Lancet 2016 被引用: 1,016