Conformationally Restricted Negamycin Derivatives Display Potent Readthrough Activity for TGA and TAG Mutations in Duchenne Muscular Dystrophy

https://pubmed.ncbi.nlm.nih.gov/38116427

Scientists have developed conformationally restricted negamycin derivatives, TCP-304 and TCP-306, that show potent readthrough activity for TGA and TAG mutations in Duchenne muscular dystrophy.