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Intrathecal administration of AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): Phase 1/2A study (strong) Finkel, R. S., Day, J. W., Darras, B. T., Kuntz, N. L., Connolly, A. M., Crawford, T. O., Butterfield, R. J., Shieh, P. B., Tennekoon, G., Iannaccone, S. T., Meriggioli, M., Ogrinc, F. G., Kavanagh, S., Kernbauer, E., Whittle, J., L'Italien, J., Kaspar, B., Sproule, D. M., Spector, S. A., Feltner, D. E., Mendell, J. R. ELSEVIER. 2019
View details for DOI 10.1016/j.jns.2019.10.322
View details for Web of Science ID 000516770701125