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Novel inhibitors of ferroptosis

Novel derivatives of oleic acid effectively inhibiting ferroptosis that could be used for treatment of Friedreich Ataxia, neurodegenerative diseases, and neuropsychiatric diseases. Problem: Ferroptosis is a non-apoptotic form of cell death characterized by the iron-dependent accumulation of toxic lipid-reactive oxygen species. Ferroptosis has been...

Published: 10/16/2020

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Up-regulators of utrophin expression for the treatment of muscular dystrophy

Small molecules targeting utrophin mRNA untranslated regions overcome post-transcriptional repression Problem: Duchenne Muscular Dystrophy (DMD) is an X-linked neuromuscular disorder that affects approximately 1 in 3500 males worldwide. DMD is caused by mutations in the DMD gene that lead to severe reduction or loss of dystrophin protein. Without...

Published: 4/28/2020

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A novel isostere to replace the carboxylic acid functional group in pharmaceuticals and other compounds

Cycloalkyl-dione derivatives with improved pharmacokinetic and toxicological properties for drug designProblem: Isosteric replacement of a specific group of atoms with a moiety with similar physicochemical properties is a strategy employed by medicinal chemists to improve the properties of biologically active compounds. For example, carboxylic acid...

Published: 3/19/2020

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