1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| oculopharyngeal muscular dystrophy | genetically modified, non-self replicating Adeno-Associated Virus serotype 9 expressing shRNA to knock down mutant PABP1 as well as a codon optimized, shRNA-insensitive, wildtype PABN1 | Orphan Designation | - |
| oculopharyngeal muscular dystrophy 1 | genetically modified, non-self replicating Adeno-Associated Virus serotype 9 expressing shRNA to knock down mutant PABP1 as well as a codon optimized, shRNA-insensitive, wildtype PABN1 | Orphan Designation | - |
| oculopharyngeal muscular dystrophy 2 | genetically modified, non-self replicating Adeno-Associated Virus serotype 9 expressing shRNA to knock down mutant PABP1 as well as a codon optimized, shRNA-insensitive, wildtype PABN1 | Orphan Designation | - |