1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Stargardt disease 3 | 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-pyrazol-1-yl) propanoic acid | Orphan Designation | - |
| Stargardt disease 4 | 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-pyrazol-1-yl) propanoic acid | Orphan Designation | - |
| severe early-childhood-onset retinal dystrophy | 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-pyrazol-1-yl) propanoic acid | Orphan Designation | - |