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Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA covalently linked to a ligand containing three N-acetylgalactosamine residues |
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Trade Name | |
Orphan Indication | Variegate porphyria |
EU Market Approval | EU |
EU Designation Date | 2016-08-29 00:00:00 |
Sponsor | Alnylam UK Limited |