Results from a phase 1 study of nusinersen (ISIS-SMN Rx) in children with spinal muscular atrophy

Claudia A. Chiriboga, Kathryn J. Swoboda, Basil T. Darras, Susan T. Iannaccone, Jacqueline Montes, Darryl C. De Vivo, Daniel A. Norris, C. Frank Bennett, Kathie M. Bishop

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