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AVXS-101 gene replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal studies clinical update (STR1VE-EU and STR1VE) 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. D., Schultz, M., Shieh, P. B., Smith, E. C., Feltner, D. E., Ouyang, H., Lavrov, A., Truncated, A., L'italien, J., Sproule, D. M., Mendell, J. R., Muntoni, F. WILEY. 2019: 224–25
View details for Web of Science ID 000474481000355