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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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Detalles Bibliográficos
Autores principales: Delnoy, Britt, Coelho, Ana I., Rubio-Gozalbo, Maria Estela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7911353/
https://www.ncbi.nlm.nih.gov/pubmed/33525536
http://dx.doi.org/10.3390/jpm11020075