2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| RPGR-related retinopathy | adeno-associated virus serotype 8 vector containing single-stranded DNA encoding the unmodified human lebercilin transgene | Orphan Designation | - |
| malignant pancreatic neoplasm | 4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl) cyclopent-2-en-1-yl] pyrimidin-2-one | Orphan Designation | - |
| muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type a, 12 | adeno-associated virus serotype 8 vector containing single-stranded DNA encoding the unmodified human lebercilin transgene | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 10 | adeno-associated virus serotype 8 vector containing single-stranded DNA encoding the unmodified human lebercilin transgene | Orphan Designation | - |