2
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Paine syndrome | Lysine acetylsalicylate injectable | Orphan Designation | - |
| TMEM165-congenital disorder of glycosylation | Lysine acetylsalicylate injectable | Orphan Designation | - |
| chromosome 1q21.1 deletion syndrome | enisoprost | Orphan Designation | - |
| chromosome 1q21.1 duplication syndrome | enisoprost | Orphan Designation | - |
| developmental and epileptic encephalopathy 119 | Lysine acetylsalicylate injectable | Orphan Designation | - |
| leukoencephalopathy with vanishing white matter 1 | Lysine acetylsalicylate injectable | Orphan Designation | - |
| pain agnosia | Lysine acetylsalicylate injectable | Orphan Designation | - |
| patellofemoral pain syndrome | Lysine acetylsalicylate injectable | Orphan Designation | - |
| thrombocytopenia-absent radius syndrome | enisoprost | Orphan Designation | - |