5
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Bietti crystalline corneoretinal dystrophy | a non-replicating recombinant adeno-associated virus vector serotype 8 delivering CYP4V2 gene | Orphan Designation | - |
| Gaucher disease | adeno-associated viral vector delivering human GBA1 gene | Orphan Designation | - |
| aromatic L-amino acid decarboxylase deficiency | a recombinant adeno-associated virus serotype 9-based vector containing an expression cassette of the human AADC and human GDNF genes | Orphan Designation | - |
| foix chavany Marie syndrome | a recombinant adeno-associated virus serotype 9-based vector containing an expression cassette of the human AADC and human GDNF genes | Orphan Designation | - |
| glutaryl-CoA dehydrogenase deficiency | adeno-associated viral vector 9 delivering human Glutaryl-CoA Dehydrogenase (GCDH) gene | Orphan Designation | - |
| hemophilia B | an adeno-associated viral vector delivering human FIX Padua gene | Orphan Designation | - |
| progressive non-fluent aphasia | a recombinant adeno-associated virus serotype 9-based vector containing an expression cassette of the human AADC and human GDNF genes | Orphan Designation | - |
| pseudo-TORCH syndrome | a non-replicating recombinant adeno-associated virus vector serotype 8 delivering CYP4V2 gene | Orphan Designation | - |