SMA – THERAPY: P.269 Impact of Continued Nusinersin treatment on Caregiver Experience and Health-Related Quality of Life in Later-onset SMA: Results From the SHINE Study

Abstract

In CHERISH, a Phase 3, randomized, double-blind, sham-controlled study (NCT02292537), nusinersen demonstrated significant and clinically meaningful improvement in motor function vs sham control in children with later-onset SMA. Within CHERISH, nusinersen was associated with reduced caregiver impact and less decline in HRQoL vs sham control. Children who completed CHERISH were eligible to enrol in the SHINE open-label extension study (NCT02594124); all participants receive nusinersen 12 mg every 4 months after a protocol amendment.