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

著者: 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. Macek, Jerry R. Mendell - The Lancet 2021 被引用: 480