1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | ALIQOPA | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | ALIQOPA | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | ALIQOPA | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | ALIQOPA | Orphan Designation | - |
| follicular lymphoma | ALIQOPA | Orphan Designation | - |
| primary adult heart tumor | ALIQOPA | Orphan Designation | - |