Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Mercuri, Eugenio; T. Darras, Basil; A. Chiriboga, Claudia; W. Day, John; Campbell, Craig; M. Connolly, Anne; T. Iannaccone, Susan; Kirschner, Janbernd; L. Kuntz, Nancy; Saito, Kayoko; B. Shieh, Perry; Tulinius, M.; Mazzone, Elena; Montes, Jacqueline; M. Bishop, Kathie; Yang, Qingqing; Foster, Richard; Gheuens, Sarah; Frank Bennett, C.; Farwell, Wildon; Schneider, Eugene; C. De Vivo, Darryl; S. Finkel, Richard

doi:10.1056/nejmoa1710504

Public

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Published in New England Journal of Medicine • Feb 14, 2018

Authors:

Eugenio Mercuri

Basil T. Darras

Claudia A. Chiriboga

Abstract

BACKGROUND: Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 ( SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). METHODS: We conducted a multicenter, double-blind, sham-controlled, phase 3 tri...

Subject

Spinal muscular atrophy

Medicine

Physical medicine and rehabilitation

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