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