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Lumasiran: First Approval
Abstract
Lumasiran (Oxlumo™) is a subcutaneously administered small interfering RNA (siRNA) targeting the mRNA for hydroxyacid oxidase 1 gene (HAO1; encodes glycolate oxidase) and was developed by Alnylam Pharmaceuticals for the treatment of primary hyperoxaluria type 1 (PH1). By silencing the gene encoding glycolate oxidase, lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly...
Paper Details
Title
Lumasiran: First Approval
Published Date
Jan 6, 2021
Journal
Volume
81
Issue
2
Pages
277 - 282
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