2
Orphan Designations
1
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | Redemplo | Orphan Designation | - |
| acute sensory ataxic neuropathy | Redemplo | Orphan Designation | - |
| aortic valve disease 1 | Redemplo | Orphan Designation | - |
| aortic valve stenosis | Redemplo | Orphan Designation | - |
| familial chylomicronemia syndrome | Redemplo | Orphan Designation | - |
| homozygous familial hypercholesterolemia | Double stranded oligomer RNA interference-based liver targeted therapeutic directed against the ANGPTL3 gene | Orphan Designation | - |