1
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Charcot-Marie-Tooth disease type 1 | synthetic double strand oligonucleotide encoding PMP22 siRNA with the sense strand modified by a DBCO residue at the 5¿-end, conjugated with a triazole SQ | Orphan Designation | - |
| Charcot-Marie-Tooth disease type 1A | synthetic double strand oligonucleotide encoding PMP22 siRNA with the sense strand modified by a DBCO residue at the 5¿-end, conjugated with a triazole SQ | Orphan Designation | - |