Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study
Titel:
Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study
Auteur:
Finkel, Richard S Chiriboga, Claudia A Vajsar, Jiri Day, John W Montes, Jacqueline De Vivo, Darryl C Bishop, Kathie M Foster, Richard Liu, Yingying Ramirez-Schrempp, Daniela Schneider, Eugene Bennett, C Frank Wong, Janice Farwell, Wildon