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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Authors
Eugenio Mercuri,Basil Darras
Claudia Chiriboga,John Day,Craig Campbell,Anne Connolly,Susan Iannaccone,Janbernd Kirschner,Nancy Kuntz,Kayoko Saito,Perry Shieh,M. Tulinius,Elena Mazzone,Jacqueline Montes,Kathie Bishop,Qingqing Yang,Richard Foster,Sarah Gheuens,C. Bennett,Wildon Farwell,Eugene Schneider,Darryl Vivo
+20 authors
,Richard FinkelPublished
Feb 14, 2018
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Abstract
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).
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