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Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG) Finkel, R., Day, J., Darras, B., Kuntz, N., Connolly, A., Crawford, T., Butterfield, R., Shieh, P., Tennekoon, G., Iannaccone, S., Ogrinc, F., Kavanagh, S., Kernbauer, E., Whittle, J., L'Italien, J., Kaspar, B., Sproule, D., Spector, S., Feltner, D., Mendell, J. PERGAMON-ELSEVIER SCIENCE LTD. 2019: S207
View details for DOI 10.1016/j.nmd.2019.06.594
View details for Web of Science ID 000487768600581