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

Publication date: April 2021Source: The Lancet Neurology, Volume 20, Issue 4Author(s): 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
Source: The Lancet Neurology - Category: Neurology Source Type: research