8
Orphan Designations
1
FDA Approvals
17
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Antley-Bixler syndrome with genital anomalies and disordered steroidogenesis | levothyroxine sodium | Orphan Designation | - |
| Stargardt disease | Echothiophate iodide | Orphan Designation | - |
| acute liver failure | Avaclyr | Orphan Designation | - |
| brain hypoxia - ischemia | phenobarbitol sodium injection | Orphan Designation | - |
| endophthalmitis | vancomycin | Orphan Designation | - |
| febrile infection-related epilepsy syndrome | Avaclyr | Orphan Designation | - |
| hepatic porphyria | Avaclyr | Orphan Designation | - |
| herpes simplex virus keratitis | Avaclyr | Orphan Designation | - |
| methemoglobinemia | methylene blue | Orphan Designation | - |
| non-herpetic acute limbic encephalitis | Avaclyr | Orphan Designation | - |
| perinatal asphyxia | phenobarbitol sodium injection | Orphan Designation | - |
| plasma cell myeloma | dexamethasone | Orphan Designation | - |
| respiratory underresponsiveness to hypoxia and hypercapnia | phenobarbitol sodium injection | Orphan Designation | - |
| retinitis pigmentosa 12 | levothyroxine sodium | Orphan Designation | - |
| sickle cell disease | naproxcinod | Orphan Designation | - |
| spondyloepiphyseal dysplasia with congenital joint dislocations | levothyroxine sodium | Orphan Designation | - |
| toxic epidermal necrolysis | Avaclyr | Orphan Designation | - |