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Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): Global pivotal phase 3 study program (STR1VE-US, STR1VE-EU, STR1VE-AP) Mercuri, E., Baranello, G., Day, J. W., Bruno, C., Corti, S., Chiriboga, C. A., Crawford, T. O., Darras, B. T., Finkel, R. S., Connolly, A. M., Iannaccone, S. T., Kuntz, N. L., Masson, R., Pena, L. M., Baldinetti, F., Schultz, M., Shieh, P. B., Smith, E. C., Saito, K., Scoto, M., Spector, S. A., Truncated, A., Sproule, D. M., Mendell, J. R., Muntoni, F. ELSEVIER. 2019
View details for DOI 10.1016/j.jns.2019.10.1338
View details for Web of Science ID 000516770700551