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Nedosiran is an antisense oligonucleotide used to treat primary hyperoxaluria type 1 in adults and high oxalate levels in children
Nedosiran is an antisense oligonucleotide used to treat primary hyperoxaluria type 1 in adults and high oxalate levels in children
Nedosiran is a double-stranded siRNA, conjugated to GalNAc aminosugar residues. After subcutaneous administration, the GalNAc-conjugated sugars bind to asialoglycoprotein receptors (ASGPR) to deliver nedosiran to hepatocytes. Nedosiran reduces levels of hepatic lactate dehydrogenase (LDH) via the degradation of LDHA messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. The reduction of hepatic LDH by nedosiran reduces the production of oxalate by the liver, thereby reducing subsequent oxalate burden.
RIVFLOZA is indicated to lower urinary oxalate levels in children 9 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function, e.g., eGFR ≥ 30 mL/min/1.73 m.
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
