The new drug not only is a boon for patients with spinal muscular atrophy, but also may spearhead the development of further chemical RNA modulators for a broad range of conditions.
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05 February 2021
A Correction to this paper has been published: https://doi.org/10.1038/s41587-021-00841-7
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Sheridan, C. First small-molecule drug targeting RNA gains momentum. Nat Biotechnol 39, 6–8 (2021). https://doi.org/10.1038/s41587-020-00788-1
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DOI: https://doi.org/10.1038/s41587-020-00788-1
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