3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 22q11.2 deletion syndrome | L-citrulline | Orphan Designation | - |
| Meckel syndrome, type 6 | ursodiol | Orphan Designation | - |
| chromosome 2q32-q33 deletion syndrome | L-citrulline | Orphan Designation | - |
| endogenous depression | L-citrulline | Orphan Designation | - |
| monosomy X | ursodiol | Orphan Designation | - |
| mosaic monosomy X | ursodiol | Orphan Designation | - |
| sickle cell disease | L-citrulline | Orphan Designation | - |