2
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Leber hereditary optic neuropathy | recombinant adeno-associated virus serotype 2 containing human mitochondrial ND1 codon optimized gene (rAAV2-ND1)Recombinant Human Adeno-Associated Virus Serotype 2 Containing Human Mitochondrial ND4 gene (rAAV2-ND4) | Orphan Designation | - |
| Leber hereditary optic neuropathy, autosomal recessive | recombinant adeno-associated virus serotype 2 containing human mitochondrial ND1 codon optimized gene (rAAV2-ND1) | Orphan Designation | - |
| Leber optic atrophy and dystonia | recombinant adeno-associated virus serotype 2 containing human mitochondrial ND1 codon optimized gene (rAAV2-ND1) | Orphan Designation | - |