3
Orphan Designations
1
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acromegaly | Palsonify | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Palsonify | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Palsonify | Orphan Designation | - |
| carcinoid syndrome | paltusotine | Orphan Designation | - |
| cataract 13 with adult I phenotype | Palsonify | Orphan Designation | - |
| classic congenital adrenal hyperplasia | atumelnant | Orphan Designation | - |
| congenital adrenal hyperplasia | atumelnant | Orphan Designation | - |
| non-classic congenital adrenal hyperplasia | atumelnant | Orphan Designation | - |
| nut midline carcinoma | Palsonify | Orphan Designation | - |