Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

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

doi:10.1056/nejmoa1710504

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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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Mercuri et al. (2018) studied this question.

synapsesocial.com/papers/69d7b281b843b2be99490721 https://doi.org/10.1056/nejmoa1710504

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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

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