3
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| classic homocystinuria | Ceprotin | Orphan Designation | - |
| factor 5 excess with spontaneous thrombosis | Ceprotin | Orphan Designation | - |
| hemophilia B | An Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua (AAV8.ss-3xCRM8-TTRFIX_R338Lopt) | Orphan Designation | - |
| overhydrated hereditary stomatocytosis | Ceprotin | Orphan Designation | - |
| protein-losing enteropathy | Ceprotin | Orphan Designation | - |
| purpura fulminans | Ceprotin | Orphan Designation | - |
| sickle cell disease | 5-hydroxymethyl-2-furfuraldehyde | Orphan Designation | - |
| thrombophilia due to thrombin defect | Ceprotin | Orphan Designation | - |