Estudo de braço único fase 3 | Onasemnogene abeparvovec para bebês pré-sintomáticos com 3 cópias de SMN2 sob risco de atrofia muscular espinal.

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial – Nature Medicine

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For infants with three copies of #SMN1 at risk for #SMA type 1, onasemnogene abeparvovec improves ventilator-free survival and nutritional/respiratory independence and allows motor development similar to healthy children without SMA. #2022SMAConferencehttps://t.co/tIsR1fByNK

— Nature Medicine (@NatureMedicine) June 17, 2022