Nusinersen versus sham control in infantile-onset spinal muscular atrophy

The trial that proved a splice switching antisense oligonucleotide could change the course of a fatal disease. Nusinersen redirects the splicing of the SMN2 gene to restore a working protein in infants with spinal muscular atrophy, dramatically improving survival and motor milestones. It became one of the first widely approved ASO drugs and a landmark for the whole modality.