4
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Myqorzo | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Myqorzo | Orphan Designation | - |
| amyotrophic lateral sclerosis | Reldesemtivtirasemtiv | Orphan Designation | - |
| cataract 13 with adult I phenotype | Myqorzo | Orphan Designation | - |
| nut midline carcinoma | Myqorzo | Orphan Designation | - |
| spinal muscular atrophy | fast skeletal muscle troponin activator | Orphan Designation | - |
| symptomatic form of hemophilia A in female carriers | Myqorzo | Orphan Designation | - |