Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

20172,220 citationsJournal Articlebronze Open Access

Authors

Richard S. Finkel · Nemours Children's Clinic

Eugenio Mercuri · Catholic University of America

Basil T. Darras · Boston Children's Hospital

Anne M. Connolly · St. Louis Children's Hospital

Nancy L. Kuntz · Lurie Children's Hospital

Janbernd Kirschner · University of Freiburg

Claudia A. Chiriboga

Abstract

Among infants with spinal muscular atrophy, those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group. Early treatment may be necessary to maximize the benefit of the drug. (Funded by Biogen and Ionis Pharmaceuticals; ENDEAR ClinicalTrials.gov number, NCT02193074 .).

Topics & Keywords

Neurogenetic and Muscular Disorders Research RNA modifications and cancer RNA Research and Splicing

Publication Details

Published in: New England Journal of Medicine

Volume 377, Issue 18, pp. 1723-1732

DOI: 10.1056/nejmoa1702752

Field-Weighted Citation Impact: 111.44

Command Palette

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Authors

Abstract

Topics & Keywords

Publication Details