1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Nijmegen breakage syndrome | Encelto | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Encelto | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Encelto | Orphan Designation | - |
| cataract 13 with adult I phenotype | Encelto | Orphan Designation | - |
| facioscapulohumeral muscular dystrophy 1 | Encelto | Orphan Designation | - |
| nut midline carcinoma | Encelto | Orphan Designation | - |