Targeting Pathological RNA in Myotonic Dystrophy (DM1): Advancing a Small Molecule with Intrinsic Activity to Treat a Systemic Disease
• Harnessing structure-based drug design to develop rSMs to normalize RNA splicing and relieve myotonia
• Optimizing the pharmacological properties of an rSM with systemic biodistribution and functional activity in disease-relevant models
• Demonstrating molecular correction and phenotypic rescue to support the translational potential of a first-in-class small molecule against DM1