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 LANCET NEUROLOGY Day, J. W., Finkel, R. S., Chiriboga, C. A., Connolly, A. M., Crawford, T. O., Darras, B. T., Iannaccone, S. T., Kuntz, N. L., Pena, L. M., Shieh, P. B., Smith, E. C., Kwon, J. M., Zaidman, C. M., Schultz, M., Feltner, D. E., Tauscher-Wisniewski, S., Ouyang, H., Chand, D. H., Sproule, D. M., Macek, T. A., Mendell, J. R. 2021; 20 (4): 284–93

View details for Web of Science ID 000630325700019