4
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Niemann-Pick disease, type C1 | Adeno-associated viral vector serotype 9 containing the human NPC1 gene | Orphan Designation | - |
| PLA2G6-associated neurodegeneration | adeno-associated viral vector serotype 9 containing the human PLA2G6 gene | Orphan Designation | - |
| SLC6A3-related dopamine transporter deficiency syndrome | adeno-associated viral vector serotype 2 containing the human SLC6A3 gene | Orphan Designation | - |
| atypical dopamine transporter deficiency syndrome | adeno-associated viral vector serotype 2 containing the human SLC6A3 gene | Orphan Designation | - |
| classic dopamine transporter deficiency syndrome | adeno-associated viral vector serotype 2 containing the human SLC6A3 gene | Orphan Designation | - |
| ornithine carbamoyltransferase deficiency | adeno-associated viral vector serotype LK03 containing the human ornithine transcarbamylase gene | Orphan Designation | - |