2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| chronic granulomatous disease | two independent HDAd vectors, in which one vector contains the integration transgenes and the other vector the CYBB therapeutic transgene | Orphan Designation | - |
| urethral obstruction sequence | GRObeta (a truncated form of the human CXC motif chemokine 2) | Orphan Designation | - |