2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Dravet syndrome | synthetic, single stranded, fully phosphorothioated 2¿-OMethyl-RNA and DNA mixmer oligonucleotide-based compound targeted against natural antisense transcripts (NATs) | Orphan Designation | - |
| urea cycle disorder | single strand 20-mer antisense oligonucleotide (ASO) targeting a CPS1 regulatory RNA with a triantennary N-acetylgalactosamine (GalNAc) ligand attached at the 3' end | Orphan Designation | - |