GALK inhibitors for classic galactosemia

Future Med Chem. 2014 Jun;6(9):1003-15. doi: 10.4155/fmc.14.43.

Abstract

Classic galactosemia is an inherited metabolic disease for which, at present, no therapy is available apart from galactose-restricted diet. However, the efficacy of the diet is questionable, since it is not able to prevent the insurgence of chronic complications later in life. In addition, it is possible that dietary restriction itself could induce negative side effects. Therefore, there is a need for an alternative therapeutic approach that can avert the manifestation of chronic complications in the patients. In this review, the authors describe the development of a novel class of pharmaceutical agents that target the production of a toxic metabolite, galactose-1-phosphate, considered as the main culprit for the cause of the complications, in the patients.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Galactokinase / antagonists & inhibitors*
  • Galactokinase / deficiency
  • Galactokinase / metabolism
  • Galactosemias / drug therapy*
  • Galactosemias / enzymology*
  • Galactosemias / metabolism
  • Humans

Substances

  • Galactokinase