3
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Kocher-debre-Semelaigne syndrome | Growth hormone releasing factor | Orphan Designation | - |
| candidiasis, familial, 1 | verteporfin | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | Tiazofurin (2-Beta-D-ribofuranosyl-4-thiazolecarboxamide) | Orphan Designation | - |
| laryngo-onycho-cutaneous syndrome | Growth hormone releasing factor | Orphan Designation | - |
| nut midline carcinoma | Growth hormone releasing factor | Orphan Designation | - |
| short-rib thoracic dysplasia 9 with or without polydactyly | verteporfin | Orphan Designation | - |