1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | WinRho SD | Orphan Designation | - |
| Sandhoff disease, adult form | WinRho SD | Orphan Designation | - |
| autoimmune thrombocytopenic purpura | WinRho SD | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | WinRho SD | Orphan Designation | - |
| multisystem inflammatory syndrome in children and adults | WinRho SD | Orphan Designation | - |
| nut midline carcinoma | WinRho SD | Orphan Designation | - |