Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

S. Finkel, Richard; Mercuri, Eugenio; T. Darras, Basil; M. Connolly, Anne; L. Kuntz, Nancy; Kirschner, Janbernd; A. Chiriboga, Claudia; Saito, Kayoko; Servais, Laurent; F. Tizzano, Eduardo; Topaloğlu, Haluk; Tulinius, M.; Montes, Jacqueline; M. Glanzman, Allan; M. Bishop, Kathie; Zhong, Zhensbao; Gheuens, Sarah; Frank Bennett, C.; Schneider, Eugene; Farwell, Wildon; C. De Vivo, Darryl

doi:10.1056/nejmoa1702752

Public

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Published in New England Journal of Medicine • Nov 1, 2017

Authors:

Richard S. Finkel

Eugenio Mercuri

Basil T. Darras

Abstract

BACKGROUND: Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased p...

Subject

Spinal muscular atrophy

Motor neuron

RNA splicing

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