2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| PGM1-congenital disorder of glycosylation | Triheptanoin | Orphan Designation | - |
| Sjogren-Larsson syndrome | Triheptanoin | Orphan Designation | - |
| disorder of glycogen metabolism | Triheptanoin | Orphan Designation | - |
| glycogen storage disease II | Triheptanoin | Orphan Designation | - |
| inherited fatty acid metabolism disorder | Triheptanoin | Orphan Designation | - |
| inherited lipid metabolism disorder | Triheptanoin | Orphan Designation | - |