1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| glycogen storage disease IX | Modified mRNA that encodes human glucose-6-phosphatase-alpha | Orphan Designation | - |
| glycogen storage disease IXc | Modified mRNA that encodes human glucose-6-phosphatase-alpha | Orphan Designation | - |
| glycogen storage disease Ib | Modified mRNA that encodes human glucose-6-phosphatase-alpha | Orphan Designation | - |