3
Orphan Designations
0
FDA Approvals
3
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wolfram syndrome | Sodium phenylbutyrate and tauroursodeoxycholic acid dihydrate | Orphan Designation | - |
| amyotrophic lateral sclerosis | Relyvrio | Orphan Designation | - |
| hyperinsulinemic hypoglycemia | avexitide | Orphan Designation | - |