Single-arm phase 3 Trial: Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy.

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

Commentary on Twitter

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