1
Orphan Designations
0
FDA Approvals
1
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| LAMA2-related muscular dystrophy | muscle-tropic derivative adeno-associated virus serotype 9 (AAV9) gene therapy product (MYO-AAV3A.CK8.GNDM.U6.sgHu) | Orphan Designation | - |