Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
Title:
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
Author:
Finkel, Richard S Chiriboga, Claudia A Vajsar, Jiri Day, John W Montes, Jacqueline De Vivo, Darryl C Yamashita, Mason Rigo, Frank Hung, Gene Schneider, Eugene Norris, Daniel A Xia, Shuting Bennett, C Frank Bishop, Kathie M