2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| anterior ischemic optic neuropathy | synthetic double-stranded siRNA oligonucleotide directed against p53 mRNA | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Teprasiran | Orphan Designation | - |
| delayed membranous cranial ossification | Teprasiran | Orphan Designation | - |
| delayed puberty, self-limited | Teprasiran | Orphan Designation | - |