6
Orphan Designations
0
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | Gamifant | Orphan Designation | - |
| Lesch-Nyhan syndrome | pegsitacase | Orphan Designation | - |
| Sandhoff disease, adult form | Gamifant | Orphan Designation | - |
| adult-onset Still disease | interleukin-1 receptor antagonist anakinra | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | Gamifant | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Gamifant | Orphan Designation | - |
| dyscalculia | interleukin-1 receptor antagonist anakinra | Orphan Designation | - |
| glycogen storage disease VII | Polyethylene glycol-modified uricase | Orphan Designation | - |
| hemophagocytic syndrome | Gamifant | Orphan Designation | - |
| neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia | Gamifant | Orphan Designation | - |
| systemic-onset juvenile idiopathic arthritis | interleukin-1 receptor antagonist anakinra | Orphan Designation | - |
| tubulointerstitial kidney disease, autosomal dominant, 2 | Polyethylene glycol-modified uricase | Orphan Designation | - |
| tumor lysis syndrome | Polyethylene glycol-modified uricase | Orphan Designation | - |
| tumor of adipose tissue | Polyethylene glycol-modified uricase | Orphan Designation | - |
| tumor of salivary gland | Polyethylene glycol-modified uricase | Orphan Designation | - |