11
Orphan Designations
5
FDA Approvals
32
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 5-fluorouracil poisoning | uridine triacetate | Orphan Designation | - |
| 5-fluorouracil toxicity | uridine triacetate | Orphan Designation | - |
| 5-hydroxytryptamine oxygenase regulator | uridine triacetate | Orphan Designation | - |
| 5-nucleotidase syndrome | uridine triacetate | Orphan Designation | - |
| Dandy-Walker malformation-postaxial polydactyly syndrome | Voraxaze | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | uridine triacetate | Orphan Designation | - |
| brain cancer | paclitaxel aqueous gel | Orphan Designation | - |
| cold agglutinin disease | uridine triacetate | Orphan Designation | - |
| congenital alveolar dysplasia | uridine triacetate | Orphan Designation | - |
| coronary artery disorder | uridine triacetate | Orphan Designation | - |
| esophageal cancer | paclitaxel aqueous gel | Orphan Designation | - |
| frostbite | Aurlumyn | Orphan Designation | - |
| gastric carcinoma | Plevitrexed | Orphan Designation | - |
| liver disease, severe congenital | Voraxaze | Orphan Designation | - |
| long QT syndrome 16 | Voraxaze | Orphan Designation | - |
| orotic aciduria | uridine triacetate | Orphan Designation | - |
| ovarian cancer | Plevitrexed | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | uridine triacetate | Orphan Designation | - |
| pediatric hepatocellular carcinoma | uridine triacetate | Orphan Designation | - |
| pediatric ovarian dysgerminoma | uridine triacetate | Orphan Designation | - |
| pediatric-onset Graves disease | uridine triacetate | Orphan Designation | - |
| ricin poisoning | Ricin Immune Fab | Orphan Designation | - |
| severe Canavan disease | Aurlumyn | Orphan Designation | - |
| severe achondroplasia-developmental delay-acanthosis nigricans syndrome | Aurlumyn | Orphan Designation | - |
| severe intellectual disability-poor language-strabismus-grimacing face-long fingers syndrome | Aurlumyn | Orphan Designation | - |
| severe neonatal-onset encephalopathy with microcephaly | Aurlumyn | Orphan Designation | - |
| stomach carcinoma in situ | Plevitrexed | Orphan Designation | - |
| systemic sclerosis | iloprost | Orphan Designation | - |
| toxic diffuse goiter | Voraxaze | Orphan Designation | - |
| toxic epidermal necrolysis | Voraxaze | Orphan Designation | - |
| toxic shock syndrome | Voraxaze | Orphan Designation | - |
| undifferentiated carcinoma of stomach | Plevitrexed | Orphan Designation | - |