5
Orphan Designations
0
FDA Approvals
16
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Combarros Calleja Leno syndrome | Lamictal | Orphan Designation | - |
| DYRK1A-related intellectual disability syndrome | Melphalan | Orphan Designation | - |
| Lennox-Gastaut syndrome | Lamictal | Orphan Designation | - |
| Pneumocystis infectious disease | Mepron | Orphan Designation | - |
| RFT1-congenital disorder of glycosylation | Mepron | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Lamictal | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Lamictal | Orphan Designation | - |
| amyloidosis, primary localized cutaneous, 1 | Mepron | Orphan Designation | - |
| anosmia for isobutyric acid | Melphalan | Orphan Designation | - |
| cystic fibrosis | Amiloride HCl solution for inhalation | Orphan Designation | - |
| metastatic malignant neoplasm in the colon | Leucovorin calcium | Orphan Designation | - |
| metastatic melanoma | Leucovorin calcium | Orphan Designation | - |
| metastatic neoplasm | Leucovorin calcium | Orphan Designation | - |
| neuroacanthocytosis | Melphalan | Orphan Designation | - |
| pneumocystosis | Mepron | Orphan Designation | - |
| primary adult heart tumor | Lamictal | Orphan Designation | - |