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Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines

[Image: see text] Not too long ago, the concept of selectively targeting mRNA with small molecules was perceived as a formidable scientific challenge. The discovery of small molecule splicing modifiers and the development of risdiplam for the treatment of spinal muscular atrophy (SMA) have firmly es...

詳細記述

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書誌詳細
出版年:ACS Med Chem Lett
主要な著者: Ratni, Hasane, Scalco, Renata S., Stephan, Alexander H.
フォーマット: Artigo
言語:Inglês
出版事項: American Chemical Society 2021
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC8201486/
https://ncbi.nlm.nih.gov/pubmed/34141064
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acsmedchemlett.0c00659
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