1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Tay-Sachs disease | diphenylcyclopenone | Orphan Designation | - |
| Wolf-Hirschhorn syndrome | diphenylcyclopenone | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | diphenylcyclopenone | Orphan Designation | - |