2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Erwinia L-asparaginase | Orphan Designation | - |
| Loeys-Dietz syndrome 4 | Quinacrine hydrochloride | Orphan Designation | - |
| anosmia for isobutyric acid | Erwinia L-asparaginase | Orphan Designation | - |
| diaphragmatic hernia 3 | Quinacrine hydrochloride | Orphan Designation | - |
| neuroacanthocytosis | Erwinia L-asparaginase | Orphan Designation | - |
| partial lipodystrophy, congenital cataracts, and neurodegeneration syndrome | Quinacrine hydrochloride | Orphan Designation | - |