Myozyme® (alglucosidase alfa)

Pompe disease or GAA deficiency (glycogen storage disease type II, GSD II, glycogenosis type II, acid maltase deficiency) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA.

Pompe disease is a rare and severely debilitating disorder that drastically reduces a person’s muscle and respiratory function. People with Pompe disease typically experience progressive muscle weakness and breathing difficulty. Symptoms can appear within a few months of birth or during childhood, adolescence or adulthood.

Patient Consent & Agreement (PCA) Form

Other Resources:

Genzyme Corporation

Myozyme.com

NORD - National Organization for Rare Diseases

Pompe Community Website

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