POMPE DISEASE DRUG PIPELINE: INVESTIGATING MARKETED AND EXPERIMENTAL TREATMENTS

Pompe Disease Drug Pipeline: Investigating Marketed and Experimental Treatments 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 excessive gly

Improving Access to Pompe Disease Treatment in Eastern Europe Pompe disease, a rare genetic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), presents significant challenges in diagnosis, treatment, and research, especially in regions like Eastern Europe. This condition leads to severe muscle weakness, respiratory issue