2
Orphan Designations
2
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Fontaine progeroid syndrome | ZOKINVY | Orphan Designation | - |
| Hutchinson-Gilford progeria syndrome | ZOKINVY | Orphan Designation | - |
| Menkes disease | Zycubo | Orphan Designation | - |
| mucopolysaccharidosis type 6, rapidly progressing | ZOKINVY | Orphan Designation | - |
| mucopolysaccharidosis type 6, slowly progressing | ZOKINVY | Orphan Designation | - |
| platelet signal processing defect | ZOKINVY | Orphan Designation | - |
| progeroid syndrome | ZOKINVY | Orphan Designation | - |