Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy Type 1 (SMA1): Pivotal phase 3 study (STR1VE) update Day, J., Chiriboga, C., Crawford, T., Darras, B., Finkel, R., Connolly, A., Iannaccone, S., Kuntz, N., Pena, L., Schultz, M., Shieh, P., Smith, E., Feltner, D., Ogrinc, F., Ouyang, H., Macek, T., Kernbauer, E., Sproule, D., Mendell, J. PERGAMON-ELSEVIER SCIENCE LTD. 2019: S183

View details for DOI 10.1016/j.nmd.2019.06.511

View details for Web of Science ID 000487768600498