1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| autosomal recessive inherited pseudoxanthoma elasticum | Recombinant adeno-associated virus type 5 vector containing codon optimized human RPGR^ORF15 protein coding gene (rAAV5-hRPGR^ORF15) | Orphan Designation | - |
| inherited retinal dystrophy | Recombinant adeno-associated virus type 5 vector containing codon optimized human RPGR^ORF15 protein coding gene (rAAV5-hRPGR^ORF15) | Orphan Designation | - |
| retinal dystrophies primarily involving Bruch's membrane | Recombinant adeno-associated virus type 5 vector containing codon optimized human RPGR^ORF15 protein coding gene (rAAV5-hRPGR^ORF15) | Orphan Designation | - |