2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Leber hereditary optic neuropathy | lenadogene nolparvovec | Orphan Designation | - |
| retinitis pigmentosa | adeno-associated viral vector serotype 2.7m8 containing the chrimsonR-tdTomato gene | Orphan Designation | - |