1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| cardiac anomalies - developmental delay - facial dysmorphism syndrome | autologous CD34+ hematopoietic stem and progenitor cells transduced with lentiviral vector containing the human DCLRE1C complementary DNA | Orphan Designation | - |
| congenital heart defects, multiple types, 8, with or without heterotaxy | autologous CD34+ hematopoietic stem and progenitor cells transduced with lentiviral vector containing the human DCLRE1C complementary DNA | Orphan Designation | - |
| heterotaxy, visceral, 1, X-linked | autologous CD34+ hematopoietic stem and progenitor cells transduced with lentiviral vector containing the human DCLRE1C complementary DNA | Orphan Designation | - |