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

Richard S. Finkel(Nemours Children's Clinic), Claudia A. Chiriboga(Columbia University Irving Medical Center), Jiri Vajsar(University of Toronto), John W Day(Stanford University), Jacqueline Montes(Columbia University Irving Medical Center), Darryl C. De Vivo(Columbia University Irving Medical Center), Mason Yamashita(Ionis Pharmaceuticals (United States)), Frank Rigo(Ionis Pharmaceuticals (United States)), Gene Hung(Ionis Pharmaceuticals (United States)), Eugene Schneider(Ionis Pharmaceuticals (United States)), Daniel A Norris(Ionis Pharmaceuticals (United States)), Shuting Xia(Ionis Pharmaceuticals (United States)), C. Frank Bennett(Ionis Pharmaceuticals (United States)), Kathie M. Bishop(Ionis Pharmaceuticals (United States))
The Lancet
December 1, 2016
10.1016/s0140-6736(16)31408-8
Cited by 1,011

Abstract

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