4
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acquired polycythemia vera | Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing 3 N-acetylgalactosamine residues | Orphan Designation | - |
| myelodysplastic syndrome | synthetic double-stranded small interfering RNA (siRNA) oligonucleotide directed against TMPRSS6 mRNA | Orphan Designation | - |
| non-immunoglobulin-mediated membranoproliferative glomerulonephritis | synthetic double-strand small interfering ribonucleic acid (siRNA) oligonucleotide drug directed against C3 messenger RNA | Orphan Designation | - |
| polycythemia | Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing 3 N-acetylgalactosamine residues | Orphan Designation | - |
| polycythemia neonatorum | Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing 3 N-acetylgalactosamine residues | Orphan Designation | - |