2
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | Synribo | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Synribo | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Synribo | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Synribo | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | Synribo | Orphan Designation | - |
| myelodysplastic syndrome | omacetaine mepesuccinate | Orphan Designation | - |
| primary adult heart tumor | Synribo | Orphan Designation | - |