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Mechanism of RNA splicing modifiers in SMA
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Nature Reviews Drug Discovery 18, 904 (2019)
doi: https://doi.org/10.1038/d41573-019-00184-8
References
Campagne, S. et al. Structural basis of a small molecule targeting RNA for a specific splicing correction. Nat. Chem. Bio. https://doi.org/10.1038/s41589-019-0384-5 (2019)