Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1.
Sander F GarrelfsYaacov FrishbergSally A HultonMichael J KorenWilliam D O'RiordanPierre CochatGeorges DeschênesHadas Shasha-LavskyJeffrey M SalandWilliam G Van't HoffDaniel G FusterDaniella MagenShabbir H MoochhalaGesa SchalkEva SimkovaJaap W GroothoffDavid J SasKristin A MeliambroJiandong LuMarianne T SweetserPushkal P GargAkshay K VaishnawJohn M GansnerTracy L McGregorJohn C Lieskenull nullPublished in: The New England journal of medicine (2021)
Lumasiran reduced urinary oxalate excretion, the cause of progressive kidney failure in PH1. The majority of patients who received lumasiran had normal or near-normal levels after 6 months of treatment. (Funded by Alnylam Pharmaceuticals; ILLUMINATE-A ClinicalTrials.gov number, NCT03681184.).