PGM1-CDG

Orpha code: 319646OMIM code: 614921

Definition

A rare, genetic, congenital disorder of glycosylation and glycogen storage disease characterized by a wide range of clinical manifestations, most commonly presenting with bifid uvula with or without cleft palate at birth, associated with growth delay, hepatopathy with elevated aminotransferase serum levels, myopathy (including exercise-related fatigue, exercise intolerance, muscle weakness), intermittent hypoglycemia, and dilated cardiomyopathy and/or cardiac arrest, due to decreased phosphoglucomutase 1 enzyme activity. Less common manifestations include malignant hyperthermia, rhabdomyolysis, and hypogonadotropic hypogonadism with delayed puberty.

Disease data
Classification

Disease

Synonyms
CDG syndrome type It
CDG1t
CDG-It
Zespół CDG typu It
CDG-It
CDG1T
Congenital disorder of glycosylation type 1t
Congenital disorder of glycosylation type It
PGM1-related congenital disorder of glycosylation
Phosphoglucomutase-1 deficiency
ORPHA code
319646
OMIM code
614921
ICD10 code
E77.8
ICD11 code
-

No additional description.

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