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
The Lancet Neurology2021Vol. 20(4), pp. 284–293
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John 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
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