1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Peters plus syndrome | 17-a-hydroxyprogesterone caporate (oral formulation) | Orphan Designation | - |
| congenital microcephaly - severe encephalopathy - progressive cerebral atrophy syndrome | 17-a-hydroxyprogesterone caporate (oral formulation) | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | 17-a-hydroxyprogesterone caporate (oral formulation) | Orphan Designation | - |