3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| methylmalonic acidemia | adeno-associated virus 9 vector expressing a functional human codon optimized cDNA encoding MMAB | Orphan Designation | - |
| nephronophthisis 1 | Adeno-associated virus pseudotype 2/5 human Nephrocystin-5 (AAV2/5-NPHP5) | Orphan Designation | - |
| nephronophthisis 3 | Adeno-associated virus pseudotype 2/5 human Nephrocystin-5 (AAV2/5-NPHP5) | Orphan Designation | - |
| nephronophthisis 4 | Adeno-associated virus pseudotype 2/5 human Nephrocystin-5 (AAV2/5-NPHP5) | Orphan Designation | - |
| propionic acidemia | Adeno-Associated Virus 9 vector expressing a wild-type cDNA encoding human Propionyl-CoA Carboxylase, subunit beta (AAV9-hPCCB) | Orphan Designation | - |