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={Richard S. Finkel and Claudia A. Chiriboga and Jiri Vajsar and John W. Day and Jacqueline Montes and Darryl C. de Vivo and Mason Yamashita and Frank Rigo and Gene Hung and Eugene Schneider and Daniel A Norris and Shuting Xia and C. Frank Bennett and Kathie M. Bishop},
  journal={The Lancet},

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