Glycogen Storage Disease Type II (Pompe)

Definition

Glycogen storage disease type II is a lysosomal storage disease characterized by acid alpha-glucosidase enzyme deficiency and characterized by myopathy and cardiomyopathy.

Symptoms

  • Hypotonia
  • Cardiomegaly
  • Respiratory Failure
  • Progressive muscle weakness

Reasons

GAA gene mutation.

Diagnosis

Enzyme activity, genetic testing.

Treatment

Enzyme replacement therapy.

Which Polyclinic Should You Apply To?

Metabolism Polyclinic

What to Do in an Emergency?

In case of respiratory failure, emergency services should be consulted.

This page is for general information purposes only.