Current and Future Treatments for Classic Galactosemia

Current and Future Treatments for Classic Galactosemia

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 burdenso...

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Bibliographic Details
Main Authors:	Britt Delnoy, Ana I. Coelho, Maria Estela Rubio-Gozalbo
Format:	Article
Language:	English
Published:	MDPI AG 2021-01-01
Series:	Journal of Personalized Medicine
Subjects:	galactosemia type 1 classic galactosemia galactose 1-phosphate uridylyltransferase (GALT) gene therapy mRNA therapy pharmacological chaperones
Online Access:	https://www.mdpi.com/2075-4426/11/2/75

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