2
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| classic homocystinuria | hydroxocobalamin | Orphan Designation | - |
| methylmalonic acidemia | hydroxocobalamin | Orphan Designation | - |
| methylmalonic acidemia due to transcobalamin receptor defect | hydroxocobalamin | Orphan Designation | - |
| methylmalonic aciduria and homocystinuria type cblF | hydroxocobalamin | Orphan Designation | - |
| methylmalonic aciduria, cblB type | hydroxocobalamin | Orphan Designation | - |