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Strauss, K.A., Farrar, M.A., Muntoni, F. et al. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nat Med 28, 1381–1389 (2022). https://doi.org/10.1038/s41591-022-01866-4
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Strauss, Kevin A; Zaidman, Craig M; and et al, "Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: The Phase III SPR1NT trial." Nature Medicine. 28, 7. 1381 - 1389. (2022).