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First small-molecule drug targeting RNA gains momentum

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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… Click to show full abstract

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.

Keywords: molecule drug; small molecule; rna; drug; first small; drug targeting

Journal Title: Nature Biotechnology
Year Published: 2021

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