4
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| RPGR-related retinopathy | antisense oligonucleotide targeting the P23H mutation of the RHO gene | Orphan Designation | - |
| cystic fibrosis | anti-sense oligonucleotide consisting of 2¿0-Me RNA with a phosphorothioate backbone | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | antisense oligonucleotide targeting the P23H mutation of the RHO gene | Orphan Designation | - |
| muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type a, 12 | antisense oligonucleotide targeting the P23H mutation of the RHO gene | Orphan Designation | - |
| retinitis pigmentosa | antisense oligonucleotide targeting exon 13 of the USH2A geneantisense oligonucleotide targeting the c.7595-2144A>G mutation in intron 40 of the USH2A gene | Orphan Designation | - |