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Read-Through Compound Prodrugs Suppressing Premature Nonsense Mutations

Case 2013-576UCLA researchers in the Department of Neurology have identified a novel prodrug to enhance the aqueous solubility of RTC13 for the treatment of Duchenne Muscular Dystrophy and other genetic disorders caused by nonsense mutations.BACKGROUNDNearly 30% of genetic disorders including Duchenne Muscular Dystrophy (DMD) are caused by nonsense...

Published: 7/17/2025 | Inventor(s): Carmen Bertoni, Jasbir Singh

Keywords(s): Cns, Gene Therapy

Category(s): Therapeutics > CNS and Neurology, Therapeutics > Gene Therapy And Editing