Nusinersen treatment of spinal muscular atrophy - a systematic review.

Abstract

Nusinersen increased survival without permanent ventilatory support in children with SMA type 1. Improvements in SMA type 2 and 3 were less evident. Better outcomes were seen in young children with a short disease duration, particularly in children receiving nusinersen before symptom onset. Newborn SMA screening may facilitate presymptomatic treatment with splice modification (nusinersen, risdiplam) or gene implantation therapy (AVXS-101, zolgensma).