2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Leber congenital amaurosis | antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA | Orphan Designation | - |
| retinitis pigmentosa | 2¿-O-(2-methoxyethyl) modified antisense oligonucleotide targeting exon 13 in the USH2A gene | Orphan Designation | - |