2
Orphan Designations
1
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| MOGS-congenital disorder of glycosylation | Filspari | Orphan Designation | - |
| focal segmental glomerulosclerosis | sparsentan | Orphan Designation | - |
| glycogen storage disease Ib | Filspari | Orphan Designation | - |
| glycogen storage disease VI | Filspari | Orphan Designation | - |
| methylmalonic aciduria and homocystinuria type cblC | Filspari | Orphan Designation | - |
| simple cryoglobulinemia | Filspari | Orphan Designation | - |