2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | Sitaxsentan Sodium | Orphan Designation | - |
| Kabuki syndrome 1 | Sitaxsentan Sodium | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | (3S)-3-[(2S)-2-({N-[2-tert-butyl)phenyl]carbamoyl}carbonylamino) propanoylamino]-4-oxo-5-(2,3,5,6-tetrafluorophenoxy) pentanoci acid | Orphan Designation | - |
| congenital anomalies of kidney and urinary tract 3 | (3S)-3-[(2S)-2-({N-[2-tert-butyl)phenyl]carbamoyl}carbonylamino) propanoylamino]-4-oxo-5-(2,3,5,6-tetrafluorophenoxy) pentanoci acid | Orphan Designation | - |
| glycogen storage disease Ib | Sitaxsentan Sodium | Orphan Designation | - |
| neuroacanthocytosis | (3S)-3-[(2S)-2-({N-[2-tert-butyl)phenyl]carbamoyl}carbonylamino) propanoylamino]-4-oxo-5-(2,3,5,6-tetrafluorophenoxy) pentanoci acid | Orphan Designation | - |