POMPE DISEASE: CAUSES, SYMPTOMS, AND THE FUTURE OF THERAPEUTIC INNOVATIONS

Pompe Disease: Causes, Symptoms, and the Future of Therapeutic Innovations Pompe disease, also referred to as Glycogen Storage Disease Type II, is a rare genetic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). This enzyme is essential for breaking down glycogen into glucose, and its absence leads to glycogen accumulat

The Struggle for Better Pompe Disease Management in Eastern Europe Pompe disease is a rare genetic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), leading to progressive muscle weakness, respiratory complications, and organ damage. These symptoms significantly impact patients' quality of life, and addressing the disea