Enzyme Replacement Therapy in Pompe Disease
| Drug | Drug Name | Drug Description |
|---|---|---|
| DB16099 | Avalglucosidase alfa | A lysosomal glycogen-specific enzyme that is used to treat patients one year of age and older with late-onset Pompe disease, or lysosomal acid alpha-glucosidase (GAA) deficiency. |
| Drug | Drug Name | Target | Type |
|---|---|---|---|
| DB16099 | Avalglucosidase alfa | Cation-independent mannose-6-phosphate receptor | target |
| Drug | Drug Name | Phase | Status | Count |
|---|