2
Orphan Designations
3
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | Gomekli | Orphan Designation | - |
| Sandhoff disease, adult form | Gomekli | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | Gomekli | Orphan Designation | - |
| congenital heart disease | Ogsiveo | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Gomekli | Orphan Designation | - |
| dermoid cyst | Ogsiveo | Orphan Designation | - |
| desmoid tumor | Ogsiveo | Orphan Designation | - |
| familial adenomatous polyposis 1 | Ogsiveo | Orphan Designation | - |
| metachromatic leukodystrophy, adult form | Ogsiveo | Orphan Designation | - |
| neurofibromatosis type 1 | Gomekli | Orphan Designation | - |
| neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia | Gomekli | Orphan Designation | - |
| tumor predisposition syndrome 3 | Ogsiveo | Orphan Designation | - |