1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| NPHP3-related Meckel-like syndrome | gylceryl tri (4-pheynlybutyrate) | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | gylceryl tri (4-pheynlybutyrate) | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | gylceryl tri (4-pheynlybutyrate) | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | gylceryl tri (4-pheynlybutyrate) | Orphan Designation | - |
| oligodendroglioma, IDH-mutant and 1p/19q-codeleted grade 2 | gylceryl tri (4-pheynlybutyrate) | Orphan Designation | - |
| oligodendroglioma, IDH-mutant and 1p/19q-codeleted, grade 3 | gylceryl tri (4-pheynlybutyrate) | Orphan Designation | - |