2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Phelan-McDermid syndrome | adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3) | Orphan Designation | - |
| galactosemia | Adeno-associated virus 2/9 expressing human GALT gene (AAV2/9-hGALT) | Orphan Designation | - |