Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of
a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by
the body's cells to break down glycogen (a stored form of sugar) within specialized
structures called lysosomes. In patients with Pompe disease, an excessive amount of
glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle,
which prevents their normal function. The objective of this expanded access study is to
provide patients with Pompe disease in the United States (US), access to alglucosidase alfa
produced from a scaled up manufacturing process for a limited time until production at this
scale is approved for commercial use by the Food and Drug Administration.
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
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