1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| congenital myasthenic syndrome 3A | Uplizna | Orphan Designation | - |
| congenital myasthenic syndrome 4C | Uplizna | Orphan Designation | - |
| congenital myasthenic syndrome 5 | Uplizna | Orphan Designation | - |
| congenital myasthenic syndrome 6 | Uplizna | Orphan Designation | - |
| myasthenia gravis | Uplizna | Orphan Designation | - |
| myasthenic syndrome, congenital, 1B, fast-channel | Uplizna | Orphan Designation | - |