1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acral dystrophic epidermolysis bullosa | Highly branched poly(beta-amino ester) complexed with a nanoplasmid containing the human COL7A1 gene | Orphan Designation | - |
| centripetalis recessive dystrophic epidermolysis bullosa | Highly branched poly(beta-amino ester) complexed with a nanoplasmid containing the human COL7A1 gene | Orphan Designation | - |
| pretibial dystrophic epidermolysis bullosa | Highly branched poly(beta-amino ester) complexed with a nanoplasmid containing the human COL7A1 gene | Orphan Designation | - |