Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
New England Journal of Medicine2018Vol. 378(7), pp. 625–635
Citations Over TimeTop 1% of 2018 papers
Eugenio Mercuri, Basil T. Darras, Claudia A. Chiriboga, John Day, Craig Campbell, Anne M. Connolly, Susan T. Iannaccone, Janbernd Kirschner, Nancy L. Kuntz, Kayoko Saito, Perry B. Shieh, M. Tulinius, Elena Mazzone, Jacqueline Montes, Kathie M. Bishop, Qingqing Yang, Richard Foster, Sarah Gheuens, C. Frank Bennett, Wildon Farwell, Eugene Schneider, Darryl C. De Vivo, Richard S. Finkel
Abstract
Among children with later-onset SMA, those who received nusinersen had significant and clinically meaningful improvement in motor function as compared with those in the control group. (Funded by Biogen and Ionis Pharmaceuticals; CHERISH ClinicalTrials.gov number, NCT02292537 .).
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