2
Orphan Designations
1
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Allan-Herndon-Dudley syndrome | 3,5-diiodothyropropionic acid | Orphan Designation | - |
| adult-onset autosomal dominant demyelinating leukodystrophy | Hemady | Orphan Designation | - |
| exercise-induced hyperinsulinism | 3,5-diiodothyropropionic acid | Orphan Designation | - |
| indolent plasma cell myeloma | Hemady | Orphan Designation | - |
| non-secretory plasma cell myeloma | Hemady | Orphan Designation | - |
| osteosclerotic plasma cell myeloma | Hemady | Orphan Designation | - |
| plasma cell myeloma | Hemady | Orphan Designation | - |
| smoldering plasma cell myeloma | Hemady | Orphan Designation | - |