CGP3466B, also known as Omigapil maleate and TCH346, is a potent inhibitor of glyceraldehyde-3-phosphate dehydrogenase (GAPDH). It has been shown to have neuroprotective activity and received U.S. FDA Fast Track Designation for the treatment of congenital muscular dystrophy in May 2016.
Inhibitor of glyceraldehyde-3-phosphate dehydrogenase (GAPDH), neuroprotective.
The glycolytic enzyme glyceraldehyde-3-phosphate dehydrogenase (GAPDH) signals during cellular stress via several post-translational modifications that change its folding properties, protein-protein interactions and sub-cellular localization. We examined GAPDH properties in acute mouse liver injury due to ethanol and/or acetaminophen (APAP) treatment.
Proceedings of the National Academy of Sciences of the United States of America, 113(5), 1417-1422 (2016-01-21)
Cocaine exerts its behavioral stimulant effects by facilitating synaptic actions of neurotransmitters such as dopamine and serotonin. It is also neurotoxic and broadly cytotoxic, leading to overdose deaths. We demonstrate that the cytotoxic actions of cocaine reflect selective enhancement of
Congenital muscular dystrophy is a distinct group of diseases presenting with weakness in infancy or childhood and no current therapy. One form, MDC1A, is the result of laminin alpha-2 deficiency and results in significant weakness, respiratory insufficiency and early death.
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