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Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Strauss, Kevin A; Farrar, Michelle A; Muntoni, Francesco et al.
2022 • In Nature Medicine, 28 (7), p. 1381-1389

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41591_2022_Article_1866.pdf

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