4
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Brooke-Spiegler syndrome | Busulfex | Orphan Designation | - |
| DYRK1A-related intellectual disability syndrome | Busulfex | Orphan Designation | - |
| Fanconi-like syndrome | Busulfex | Orphan Designation | - |
| Nijmegen breakage syndrome | Busulfex | Orphan Designation | - |
| active tuberculosis | 5-{[(3R,4R)-1-(4-chloro-2,6-difluorophenyl)-3,4-dihydroxypiperidin-4-yl]methoxy}-8-fluoro-3,4-dihydroquinolin-2(1H)-one | Orphan Designation | - |
| autosomal dominant polycystic kidney disease | Jynarque | Orphan Designation | - |
| hematologic disorder | Busulfex | Orphan Designation | - |
| neuroacanthocytosis | Busulfex | Orphan Designation | - |
| pulmonary tuberculosis | (R)-2-methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole | Orphan Designation | - |