1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| post 5-alpha-reductase inhibitors treatment syndrome | CD34+ cells that have been transduced in vitro with a lentiviral vector carrying a codon-optimized sequence for expressing the wild type RPS19 protein | Orphan Designation | - |
| post-treatment Lyme disease syndrome | CD34+ cells that have been transduced in vitro with a lentiviral vector carrying a codon-optimized sequence for expressing the wild type RPS19 protein | Orphan Designation | - |
| treatment-refractory schizophrenia | CD34+ cells that have been transduced in vitro with a lentiviral vector carrying a codon-optimized sequence for expressing the wild type RPS19 protein | Orphan Designation | - |