Pompe Disease Epidemiology Forecast to 2027 : Analysis by Disease Type and Current Market Trends
Pompe disease is a rare multisystem disorder caused by pathogenic variations in the GAA gene, which contains the information for production and function of a protein called acid alpha-glucosidase (GAA). Because of the shortage of this protein (an enzyme), a complex sugar named glycogen cannot be degraded to a simple sugar like glucose. This causes the glycogen to accumulate in all kinds of tissues, but primarily in skeletal muscle, smooth muscle, and cardiac muscle, where it causes damage to tissue...
View full press release