Pompe's Disease
Also known as: Pompe Disease / Cardiac glycogenosis / Acid maltase deficiency / Glycogen storage disease type II / Type II glycogen storage disease
| Drug | Drug Name | Drug Description |
|---|---|---|
| DB01272 | Alglucosidase alfa | An acid alpha- glucosidase (GAA) derivative used as an enzyme replacement therapy for the treatment of Pompe disease in infants and pediatric patients caused by GAA deficiency. |
| Drug | Drug Name | Target | Type |
|---|---|---|---|
| DB01272 | Alglucosidase alfa | Cation-dependent mannose-6-phosphate receptor | target |
| DB01272 | Alglucosidase alfa | Glycogen | target |