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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...
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| Published in: | ACS Med Chem Lett |
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| Main Authors: | , , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
American Chemical
Society
2021
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| Online Access: | 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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