February 14, 2018Open Access
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Key Points
Key points are not available for this paper at this time.
Abstract
Among children with later-onset SMA, those who received nusinersen had significant and clinically meaningful improvement in motor function as compared with those in the control group. (Funded by Biogen and Ionis Pharmaceuticals; CHERISH ClinicalTrials.gov number, NCT02292537 .).