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Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

Nature Neuroscience volume 20, pages 497–499 (2017)Cite this article

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Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.

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Figure 1: Alteration of SMN2 splicing by an ASO.

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Authors and Affiliations

  1. David R. Corey is in the Departments of Pharmacology and Biochemistry, UT Southwestern, Dallas, Texas, USA.,

    David R Corey

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  1. David R Corey
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Correspondence to David R Corey.

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Competing interests

D.R.C. receives licensing income from Ionis Pharmaceuticals for an unrelated discovery.

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Corey, D. Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy. Nat Neurosci 20, 497–499 (2017). https://doi.org/10.1038/nn.4508

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