SMA – THERAPY: P.257 Longer-term treatment with nusinersen: Results in later-onset spinal muscular atrophy from the SHINE study

Abstract

We present results from the SHINE open-label extension study (NCT02594124) 27 August 2019 interim data cut for participants with later-onset SMA. Following protocol amendment, all participants transitioned to the Modified Maintenance Dosing Regimen (MMDR; 12 mg nusinersen every 4 months). 83 participants (81 with MMDR Day 1 data) treated with nusinersen and 42 with sham-procedure in CHERISH transitioned to SHINE. Median (range) age in years at MMDR Day 1 was 6.62 (4.1–11.5) for those who received nusinersen in CHERISH/SHINE and 5.98 (4.0–10.2) for those who received sham-procedure in CHERISH/nusinersen in SHINE.