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Glycogen storage disease due to phosphoglycerate kinase 1 deficiency

ORPHA:713· ICD-10 E74.0

Definition

A rare inborn errors of metabolism characterized by variable combinations of non-spherocytic hemolytic anemia, myopathy, and various central nervous system abnormalities.

Prevalence
<1 / 1 000 000
Inheritance
X-linked recessive
Age of onset
Adolescent, Adult, Childhood