1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | PEG-glucocerebrosidase | Orphan Designation | - |
| anosmia for isobutyric acid | PEG-glucocerebrosidase | Orphan Designation | - |
| neuroacanthocytosis | PEG-glucocerebrosidase | Orphan Designation | - |