2
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | Topamax | Orphan Designation | - |
| Lennox-Gastaut syndrome | Topamax | Orphan Designation | - |
| Sandhoff disease | Cladribine | Orphan Designation | - |
| aortic valve disease 1 | Topamax | Orphan Designation | - |
| aortic valve stenosis | Topamax | Orphan Designation | - |
| calcific aortic disease with immunologic abnormalities, familial | Topamax | Orphan Designation | - |
| glycogen storage disease II | Cladribine | Orphan Designation | - |
| neurodegeneration with brain iron accumulation 2A | Cladribine | Orphan Designation | - |