Associated Papers
Coagulation abnormalities and vascular complications are common in PGM1-CDG.
Radenkovic S et al.
Molecular Genetics and Metabolism, 2024
38968673 (PubMed )
Supplement: NIHMS2125712-supplement-Supplementary_material.xlsx › Sup_Tab_1
AAV-based gene therapy prevents and halts the progression of dilated cardiomyopathy in a mouse model of phosphoglucomutase 1 deficiency (PGM1-CDG).
Balakrishnan B et al.
Translational Research : the Journal of Laboratory and Clinical Medicine, 2023
Novel insights into the phenotype and long-term D-gal treatment in PGM1-CDG: a case series.
Radenkovic S et al.
Therapeutic Advances in Rare Disease, 0
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