Pompe Disease (PD), also known as glycogen storage disease II, is a recessive genetic metabolic disorder caused by a deficiency in the enzyme ?-glucosidase (GAA). GAA is normally responsible for hydrolyzing ?-1, 4 and ?-1, 6 linkages in various carbohydrates, including maltose, isomaltose, and glycogen. The absence or deficiency of GAA results in excessive storage of glycogen in cellular lysosomes. This inhibition can lead to cellular stress and injury. In PD, cardiac muscle tissue is severely affected alongside neurons in both the autonomic nervous system (ANS) and central nervous system (CNS).


  • GlobalData’s Pompe Disease - Opportunity Assessment and Forecast combines data from the Pharma Intelligence Center with in-house analyst expertise to provide a competitive assessment of the disease marketplace.

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