Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines
Autor: | Hasane Ratni, Renata S. Scalco, Alexander Stephan |
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Rok vydání: | 2020 |
Předmět: |
Drug
010405 organic chemistry business.industry media_common.quotation_subject Organic Chemistry Computational biology Molecular Pharmacology 01 natural sciences Biochemistry Small molecule 0104 chemical sciences 010404 medicinal & biomolecular chemistry Transformative learning Blueprint Drug Discovery RNA splicing Screening method Medicine Target mrna business media_common |
Zdroj: | ACS Med Chem Lett |
ISSN: | 1948-5875 |
Popis: | [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 established proof of concept for this exciting new platform and transformed a scientific curiosity into a viable technology to target disease. Today, several approaches to target mRNA with small molecules, supported by biophysical and screening methods, are in place to deliver new drugs with high therapeutic relevance. |
Databáze: | OpenAIRE |
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