Small-Molecule Inhibitors of Human Galactokinase for the Treatment of Galactosemia and Cancers
Lead Inventor: Matthew Boxer (NCATS)
Ref. No.: E-240-2011/0
Abstract: Lactose, found in dairy products and other foods, is comprised of two simple sugars, glucose and galactose. In galactosemia, where galactose is not properly metabolized, build-up of toxic compounds, such as galactose-1-phosphate, can lead to liver disease, renal failure, cataracts, brain damage, and even death if this disorder is left untreated. Currently, the only treatment for galactosemia is elimination of lactose and galactose from the diet, but in some cases this is not sufficient to avoid long-term complications from the disorder.