2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Becker muscular dystrophy | adeno-associated virus transgene of follistatin | Orphan Designation | - |
| Duchenne muscular dystrophy | adeno-associated virus transgene of follistatin | Orphan Designation | - |
| Erb palsy | adeno-associated virus transgene of follistatin | Orphan Designation | - |
| inclusion body myositis | Adeno-associated virus delivered transgene of follistatin | Orphan Designation | - |