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

作者：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 · 发表于：The Lancet · 年份：2016 · DOI：10.1016/s0140-6736(16)31408-8 · 被引用次数：1044 · 研究领域：Neurogenetic and Muscular Disorders Research、Amyotrophic Lateral Sclerosis Research、Craniofacial Disorders and Treatments