2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Stargardt disease | ABCA4 DNA nanoparticles | Orphan Designation | - |
| Stargardt disease 4 | ABCA4 DNA nanoparticles | Orphan Designation | - |
| cystic fibrosis | Plasmid DNA vector expressing cystic fibrosis transmembrane gene | Orphan Designation | - |
| severe early-childhood-onset retinal dystrophy | ABCA4 DNA nanoparticles | Orphan Designation | - |