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Search Results - donna huryn

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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 appropriate...
Published: 4/28/2020   |   Inventor(s): Tejvir Khurana, Donna Huryn
Keywords(s): Drug Target, Genetic Disorders, Musculoskeletal
Category(s): Therapeutics & Vaccines
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 design Problem: 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...
Published: 3/19/2020   |   Inventor(s): Amos Smith, Donna Huryn, Carlo Ballatore, Virginia Lee, John Trojanowski
Keywords(s):  
Category(s): Chemical Processes and Synthesis, Materials, Therapeutics & Vaccines