Dad finds life-saving treatment for deadly disease

Pompe disease, also known as Glycogen storage disease type 2 is an inherited metabolic disorder caused by an inborn lack of the enzyme acid alpha-glucosidase (also known as acid maltase), which is necessary to break down glycogen, a substance that is a source of energy for the body. This enzyme deficiency causes excess amounts of glycogen to accumulate in the lysosomes, which are structures within cells that break down waste products within the cell. This accumulation of glycogen in certain tissues, especially muscles, impairs their ability to function normally. Glycogen storage disease type 2 is a single disease continuum with variable rates of disease progression.

In 1996, John Crowley’s two youngest children were given what doctors considered a death sentence when they were diagnosed with a rare, fatal illness called Pompe disease. So he came up with a plan to save their lives