Frequent feeding is usually used to manage hypoglycemia in PGM1-CDG and can improve some other aspects of PGM1-CDG. Unlike most CDG, PGM1-CDG is treatable with oral D-galactose supplementation. (D-Gal). It was shown that D-Gal is able to improve coagulation and liver abnormalities as well as the phenotype severity in PGM1-CDG. However, D-Gal is not able to treat cardiac dysfunction in PGM1-CDG, the most severity phenotypic presentation. Hence, there is an urgent need to develop novel therapies that would directly target cardiac presentation in this disorder.
PGM1