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Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update Day, J. W., Chiriboga, C. A., Crawford, T. O., Darras, B. T., Finkel, R. S., Connolly, A. M., Iannaccone, S. T., Kuntz, N. L., Pena, L. M., Schultz, M., Shieh, P. B., Smith, E. C., Farrar, M., Feltner, D. E., Ogrinc, F. G., Macek, T. A., Kernbauer, E., Muehring, L. M., L'Italien, J., Sproule, D. M., Kaspar, B. K., Mendell, J. R. BMJ PUBLISHING GROUP. 2019
View details for DOI 10.1136/jnnp-2019-anzan.58
View details for Web of Science ID 000478895700059