Remove Clinical Trials Remove Gene Silencing Remove Genotype Remove RNA
article thumbnail

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1.