3
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| X-linked cerebral adrenoleukodystrophy | potassium 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo- 5-phenyl-6,7-dihydrothieno[2, 3-b]pyridin-4-olate hydrate | Orphan Designation | - |
| X-linked dominant hypophosphatemic rickets | potassium 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo- 5-phenyl-6,7-dihydrothieno[2, 3-b]pyridin-4-olate hydrate | Orphan Designation | - |
| adrenoleukodystrophy | potassium 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo- 5-phenyl-6,7-dihydrothieno[2, 3-b]pyridin-4-olate hydrate(R)-5-({4-[2-(5-ethyl-2-pyridyl)ethoxy]phenyl}methyl)-(5-2H)-1,3-thiazolidine-2,4-dione hydrochloride | Orphan Designation | - |
| autosomal dominant polycystic kidney disease | potassium 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo- 5-phenyl-6,7-dihydrothieno[2, 3-b]pyridin-4-olate hydrate | Orphan Designation | - |