1
Orphan Designations
0
FDA Approvals
1
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| beta-thalassemia HBB/LCRB | genetically modified CD34+ hematopoietic stem cells using a lentiviral vector carrying the beta A-T87Q-globin gene | Orphan Designation | - |