Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

John W. Day, Richard S. Finkel, Claudia A. Chiriboga, Anne M. Connolly, Thomas O. Crawford, Basil T. Darras, Susan T. Iannaccone, Nancy L. Kuntz, Loren D.M. Peña, Perry B. Shieh, Edward C. Smith, Jennifer M. Kwon, Craig M. Zaidman, Meredith Schultz, Douglas E. Feltner, Sitra Tauscher-Wisniewski, Haojun Ouyang, Deepa H. Chand, Douglas M. Sproule, Thomas A. MacekJerry R. Mendell

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