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