1
Orphan Designations
0
FDA Approvals
1
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| congenital disorder of deglycosylation 1 | Adeno-associated virus serotype 9 virus particle containing viral DNA that includes an expression cassette containing the human NGLY1 gene coding sequence | Orphan Designation | - |