3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | indoximod | Orphan Designation | - |
| Finnish type amyloidosis | indoximod | Orphan Designation | - |
| creatine transporter deficiency | cyclocreatine | Orphan Designation | - |
| isolated growth hormone deficiency type IA | ibutamoren mesylate | Orphan Designation | - |
| isolated growth hormone deficiency type IB | ibutamoren mesylate | Orphan Designation | - |
| isolated growth hormone deficiency type II | ibutamoren mesylate | Orphan Designation | - |
| lysinuric protein intolerance | indoximod | Orphan Designation | - |