4
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Char syndrome | Uridine 5'-triphosphate | Orphan Designation | - |
| aldosterone-producing adenoma with seizures and neurological abnormalities | Uridine 5'-triphosphate | Orphan Designation | - |
| ciliary dyskinesia, primary, 40 | Uridine 5'-triphosphate | Orphan Designation | - |
| cystic fibrosis | p1-(uridine 5'-)-p4-(2'-deoxycytidine 5'-) tetraphosphate, tetrasodium saltP1, P4-Di(uridine 5'-tetraphosphate), tetrasodium saltUridine 5'-triphosphate | Orphan Designation | - |