3
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | FluorouracilMethotrexate | Orphan Designation | - |
| Wolf-Hirschhorn syndrome | Methotrexate | Orphan Designation | - |
| acute myeloblastic leukemia with maturation | Novantrone | Orphan Designation | - |
| acute myeloid leukemia | Novantrone | Orphan Designation | - |
| acute myeloid leukemia with minimal differentiation | Novantrone | Orphan Designation | - |
| anosmia for isobutyric acid | Fluorouracil | Orphan Designation | - |
| double nail for fifth toe | Methotrexate | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Methotrexate | Orphan Designation | - |
| neuroacanthocytosis | Fluorouracil | Orphan Designation | - |
| osteosarcoma | Methotrexate | Orphan Designation | - |