3
Orphan Designations
4
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Acetadote | Orphan Designation | - |
| Wolf-Hirschhorn syndrome | Acetadote | Orphan Designation | - |
| acute liver failure | N-acetylcysteine | Orphan Designation | - |
| cardiomyopathy | Ifetroban | Orphan Designation | - |
| double nail for fifth toe | Acetadote | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Acetadote | Orphan Designation | - |