4
Orphan Designations
1
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DK1-congenital disorder of glycosylation | Tarpeyo | Orphan Designation | - |
| Fanconi-like syndrome | liposomal busulfan | Orphan Designation | - |
| Nijmegen breakage syndrome | liposomal busulfan | Orphan Designation | - |
| autoimmune hepatitis | budesonide | Orphan Designation | - |
| cardiofaciocutaneous syndrome 1 | Tarpeyo | Orphan Designation | - |
| developmental and epileptic encephalopathy, 2 | Tarpeyo | Orphan Designation | - |
| glycogen storage disease VI | Tarpeyo | Orphan Designation | - |
| hematologic disorder | liposomal busulfan | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | Tarpeyo | Orphan Designation | - |
| neurodegeneration, childhood-onset, with ataxia, tremor, optic atrophy, and cognitive decline | Tarpeyo | Orphan Designation | - |
| primary biliary cholangitis | budesonide | Orphan Designation | - |