1
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| alopecia-intellectual disability syndrome 2 | 4-aminosalicylic acid | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | 4-aminosalicylic acid | Orphan Designation | - |