2
Orphan Designations
1
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | Rytelo | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Rytelo | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Rytelo | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Rytelo | Orphan Designation | - |
| myelodysplastic syndrome | Rytelo | Orphan Designation | - |
| myelofibrosis | imetelstat | Orphan Designation | - |
| primary adult heart tumor | Rytelo | Orphan Designation | - |