4
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Dandy-Walker malformation-postaxial polydactyly syndrome | pegylated granulocyte colony stimulating factor | Orphan Designation | - |
| acquired polycythemia vera | Besremi | Orphan Designation | - |
| cataract 13 with adult I phenotype | Besremi | Orphan Designation | - |
| essential thrombocythemia | pegylated proline interferon alpha-2b | Orphan Designation | - |
| liver disease, severe congenital | pegylated granulocyte colony stimulating factor | Orphan Designation | - |
| long QT syndrome 16 | pegylated granulocyte colony stimulating factor | Orphan Designation | - |
| myelofibrosis | pegylated proline interferon alpha-2b | Orphan Designation | - |
| nut midline carcinoma | Besremi | Orphan Designation | - |