2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Charcot-Marie-Tooth disease type 1A | AAV-based engineered microRNA targeting conserved regions on the human PMP22 transcript | Orphan Designation | - |
| facioscapulohumeral muscular dystrophy | AAV-based gene therapy expressing artificial microRNA targeting the human DUX4 open reading frame | Orphan Designation | - |