Nusinersen versus sham control in infantile-onset spinal muscular atrophy
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, et al. · New England Journal of Medicine, 2017
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, et al.
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.
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