2
Orphan Designations
1
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ATTRV30M amyloidosis | TEGSEDI™ | Orphan Designation | - |
| agenesis of the corpus callosum with peripheral neuropathy | TEGSEDI™ | Orphan Designation | - |
| familial amyloid neuropathy | TEGSEDI™ | Orphan Designation | - |
| familial chylomicronemia syndrome | volanesorsen sodium, apolipotrotein C-III antisense oligonucleotide | Orphan Designation | - |
| hereditary spastic paraplegia 11 | TEGSEDI™ | Orphan Designation | - |
| hereditary spastic paraplegia 61 | TEGSEDI™ | Orphan Designation | - |
| polyneuropathy-hand defect syndrome | TEGSEDI™ | Orphan Designation | - |