1
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | INREBIC | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | INREBIC | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | INREBIC | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | INREBIC | Orphan Designation | - |
| primary adult heart tumor | INREBIC | Orphan Designation | - |
| primary myelofibrosis | INREBIC | Orphan Designation | - |
| secondary early-onset glaucoma | INREBIC | Orphan Designation | - |
| secondary hypertrophic osteoarthropathy | INREBIC | Orphan Designation | - |
| secondary vasculitis | INREBIC | Orphan Designation | - |