B.01 AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update by Day, JW, Chiriboga, CA, Crawford, TO, Darras, BT, Finkel, RS, Connolly, AM, Iannaccone, ST, Kuntz, NL, Pena, LD, Schultz, M, Shieh, PB, Smith, EC, Feltner, DE, Ogrinc, FG, Macek, TA, Kernbauer, E, Muehring, LM, L’Italien, J, Sproule, DM, Kaspar, BK, Mendell, JR

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