2
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| anosmia for isobutyric acid | Valstar | Orphan Designation | - |
| bladder carcinoma in situ | Valstar | Orphan Designation | - |
| double nail for fifth toe | Valstar | Orphan Designation | - |
| familial hypocalciuric hypercalcemia 3 | Disodium clodronate tetrahydrate | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Valstar | Orphan Designation | - |
| lethal osteosclerotic bone dysplasia | Disodium clodronate tetrahydrate | Orphan Designation | - |
| modifier, X-linked, for Neurofunctional defects | Valstar | Orphan Designation | - |
| spatial visualization, aptitude for | Valstar | Orphan Designation | - |
| tumoral calcinosis, hyperphosphatemic, familial, 1 | Disodium clodronate tetrahydrate | Orphan Designation | - |