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Article

Current and Future Treatments for Classic Galactosemia

28 Jan 2021

Abstract excerpt

Type I (classic) galactosemia, galactose 1-phosphate uridylyltransferase (GALT)-deficiency is a hereditary disorder of galactose metabolism. The current therapeutic standard of care, a galactose-restricted diet, is effective in treating neonatal complications but is inadequate in preventing burdensome complications. The development of several animal models of classic galactosemia that (partly) mimic the...

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PMID: 33525536
DOI: 10.3390/jpm11020075

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