1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| mitochondrial DNA depletion syndrome 20 (mngie type) | recombinant thymidine phosphorylase encapsulated with autologous erythrocytes | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 4b | recombinant thymidine phosphorylase encapsulated with autologous erythrocytes | Orphan Designation | - |
| mitochondrial neurogastrointestinal encephalomyopathy | recombinant thymidine phosphorylase encapsulated with autologous erythrocytes | Orphan Designation | - |