2
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Beemer-Langer syndrome | TOBI | Orphan Designation | - |
| Mounier-Kuhn syndrome | TOBI | Orphan Designation | - |
| Nezelof syndrome | TOBI | Orphan Designation | - |
| aflatoxin-related hepatocellular carcinoma | silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| brain-lung-thyroid syndrome | TOBI | Orphan Designation | - |
| congenital lethal erythroderma | silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| cystic fibrosis | TOBI | Orphan Designation | - |
| malignant carotid body paraganglioma | silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| post 5-alpha-reductase inhibitors treatment syndrome | silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| post-treatment Lyme disease syndrome | silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| treatment-refractory schizophrenia | silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| trichothiodystrophy | TOBI | Orphan Designation | - |