3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| leukocyte adhesion deficiency 1 | L-fucose | Orphan Designation | - |
| leukocyte adhesion deficiency 3 | L-fucose | Orphan Designation | - |
| leukocyte adhesion deficiency type II | L-fucose | Orphan Designation | - |
| multiple acyl-CoA dehydrogenase deficiency | D,L-3-hydroxybutyrate | Orphan Designation | - |
| ornithine carbamoyltransferase deficiency | L-Citrulline | Orphan Designation | - |