The movie “Extraordinary Measures” starring Harrison Ford and Brendan Fraser recounts the true story of one family’s search a treatment for Pompe Disease. Pompe Disease is a neuromuscular disorder that effects less than 10,000 people in the world. Today the lysosomal storage disorder can be treated with Myozyme. People living with Pompe disease lack the enzyme alpha-glucosidase which is used to process glycogen – the body’s way of storing sugar for energy. Without alpha-glucosidase glycogen builds up in the body and skeletal muscles and the heart are the two most effected areas by the excess glycogen.
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