4
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Friedreich ataxia | recombinant adeno-associated virus serotype 8 expressing codon optimized human frataxin gene cDNA under the control of a ubiquitous hPGK promoter (rAAV8-hPGK-hFXNco) | Orphan Designation | - |
| X-linked myotubular myopathy | a nonreplicating recombinant adeno-associated virus gene therapy vector expressing human MTM1 genenon-replicating adeno-associated viral vector, serotype 8, expressing human myotubularin gene | Orphan Designation | - |
| glycogen storage disease II | recombinant adeno-associated viral vector serotype 8 encoding human acid alpha-glucosidase | Orphan Designation | - |