1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Bicervical bicornuate uterus with patent cervix and vagina | Two AAV vectors, the first expressing the N- and the second expressing the C-region of the ATP-binding cassette subfamily A member 4 (ABCA4) protein fused to N- or C-terminal split-intein sequences | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Two AAV vectors, the first expressing the N- and the second expressing the C-region of the ATP-binding cassette subfamily A member 4 (ABCA4) protein fused to N- or C-terminal split-intein sequences | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | Two AAV vectors, the first expressing the N- and the second expressing the C-region of the ATP-binding cassette subfamily A member 4 (ABCA4) protein fused to N- or C-terminal split-intein sequences | Orphan Designation | - |