Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study (S25.001)

To present interim results from the ongoing NURTURE study (NCT02386553) examining efficacy/safety of intrathecal nusinersen, initiated prior to symptom onset, in infants most likely to develop SMA Type I/II.