1
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| PGM1-congenital disorder of glycosylation | Temodar | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Temodar | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Temodar | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Temodar | Orphan Designation | - |
| autosomal recessive osteopetrosis 2 | Temodar | Orphan Designation | - |
| autosomal recessive osteopetrosis 4 | Temodar | Orphan Designation | - |
| diaphyseal medullary stenosis-bone malignancy syndrome | Temodar | Orphan Designation | - |
| intellectual developmental disorder, autosomal recessive 83 | Temodar | Orphan Designation | - |
| pheochromocytoma/paraganglioma syndrome 4 | Temodar | Orphan Designation | - |
| primary adult heart tumor | Temodar | Orphan Designation | - |