1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Tay-Sachs disease | Recombinant adeno-associated virus serotype 2/1 vector encoding human beta-hexosaminidase alpha & beta subunits (rAAV2/1 Hex alpha & beta) | Orphan Designation | - |
| Tay-Sachs disease, B1 variant | Recombinant adeno-associated virus serotype 2/1 vector encoding human beta-hexosaminidase alpha & beta subunits (rAAV2/1 Hex alpha & beta) | Orphan Designation | - |
| Tay-Sachs disease, b variant, juvenile form | Recombinant adeno-associated virus serotype 2/1 vector encoding human beta-hexosaminidase alpha & beta subunits (rAAV2/1 Hex alpha & beta) | Orphan Designation | - |