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The late-onset form of Pompe disease

Publication at First Faculty of Medicine |
2021

Abstract

A case report is presented of a female patient with adult-onset Pompe disease (GSD II). The patient was diagnosed when 21 years old and is now being successfully treated with replacement therapy.

The author provides an overview of the current knowledge on the aetiopathogenesis, classification, diagnostic procedures, and treatment options of this congenital disease.