1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | Cablivi | Orphan Designation | - |
| Sandhoff disease, adult form | Cablivi | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | Cablivi | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Cablivi | Orphan Designation | - |
| neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia | Cablivi | Orphan Designation | - |
| thrombotic thrombocytopenic purpura | Cablivi | Orphan Designation | - |