GALK inhibitors for classic galactosemia

Kent Lai, Matthew B. Boxer, Anna Marabotti

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)1003-1015
Number of pages13
JournalFuture Medicinal Chemistry
Issue number9
Publication statusPublished - 2014

ASJC Scopus subject areas

  • Drug Discovery
  • Pharmacology
  • Molecular Medicine
  • Medicine(all)


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