2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| X-linked retinoschisis | Engineered adeno-associated virus spreading capsid encapsulating a human retinoschisin (hRS1) transgene | Orphan Designation | - |
| muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type a, 12 | Adeno-associated virus serotype 5 encoding human retinal guanylate cyclase 1 gene (AAV5-GUCY2D) | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 10 | Adeno-associated virus serotype 5 encoding human retinal guanylate cyclase 1 gene (AAV5-GUCY2D) | Orphan Designation | - |
| renal hypomagnesemia 3 | Adeno-associated virus serotype 5 encoding human retinal guanylate cyclase 1 gene (AAV5-GUCY2D) | Orphan Designation | - |