1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| NPHP3-related Meckel-like syndrome | (6R,S)5,10-methylene-tetrahydrofolic acid | Orphan Designation | - |
| pancreatic hypoplasia-diabetes-congenital heart disease syndrome | (6R,S)5,10-methylene-tetrahydrofolic acid | Orphan Designation | - |
| thrombocytopenia-absent radius syndrome | (6R,S)5,10-methylene-tetrahydrofolic acid | Orphan Designation | - |