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Article

Diverse targets of SMN2 -directed splicing-modulating small molecule therapeutics for spinal muscular atrophy

29 Mar 2023

Abstract excerpt

Designing an RNA-interacting molecule that displays high therapeutic efficacy while retaining specificity within a broad concentration range remains a challenging task. Risdiplam is an FDA-approved small molecule for the treatment of spinal muscular atrophy (SMA), the leading genetic cause of infant mortality. Branaplam is another small molecule which has undergone clinical trials. The therapeutic merit of both...

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PMID: 37026480
DOI: 10.1093/nar/gkad259

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