5
Orphan Designations
0
FDA Approvals
14
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Char syndrome | Dacogen | Orphan Designation | - |
| Tourette syndrome | Abilify | Orphan Designation | - |
| acute myeloid leukemia | decitabine | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Dacogen | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | decitabine | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | Dacogen | Orphan Designation | - |
| myelodysplastic syndrome | Dacogen | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | Dacogen | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Abilify | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Abilify | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Abilify | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Abilify | Orphan Designation | - |
| pediatric-onset Graves disease | Abilify | Orphan Designation | - |
| sickle cell disease | decitabine | Orphan Designation | - |