1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| maternally-inherited Leigh syndrome | modified recombinant mitochondrial transcription factor A (TFAM) containing the mitochondrial transduction domain | Orphan Designation | - |
| maternally-inherited diabetes and deafness | modified recombinant mitochondrial transcription factor A (TFAM) containing the mitochondrial transduction domain | Orphan Designation | - |
| maternally-inherited mitochondrial dystonia | modified recombinant mitochondrial transcription factor A (TFAM) containing the mitochondrial transduction domain | Orphan Designation | - |