Recent Study: Pompe Disease Market in the US 2015-2019
Pompe disease, a type of LSD, is caused by mutations in the GAA gene. These mutations inhibit the mechanism of action of GAA, an enzyme responsible for catabolizing glycogen, resulting in accumulation of glycogen in the lysosomes of the cells. The buildup of glycogen in certain organs and tissues, especially in the muscles, hinders the normal functions of the organs. Pompe disease can be further classified as infantile-onset and adult-onset Pompe disease. As the disease is an autosomal recessive hereditary...
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