1
Orphan Designations
0
FDA Approvals
1
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| phenylketonuria | recombinant lentivirus vector expressing the human phenylalanine hydroxylase (PAH) gene under control of a liver-specific promoter | Orphan Designation | - |