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Inclisiran for the treatment of heterozygous familial hypercholesterolemia

New England Journal of Medicine Mar 24, 2020

Raal FJ, Kallend D, Ray KK, et al. - This study seeks to explore the utility and safety of inclisiran for the treatment of heterozygous familial hypercholesterolemia. Researchers designed a phase 3, double-blind trial to included a total of 482 adults who had heterozygous familial hypercholesterolemia. Individuals were allocated randomly in 1:1 ration to receive subcutaneous injections of inclisiran sodium (at a dose of 300 mg) or matching placebo on days 1, 90, 270, and 450. The primary outcomes included the percent change from baseline in the LDL cholesterol level on day 510 and the time-adjusted percent change from baseline in the LDL cholesterol level between day 90 and day 540. Compared with placebo, it was noted that inclisiran had significantly lower levels of LDL cholesterol, with an infrequent dosing regimen and an acceptable safety profile among adults with heterozygous familial hypercholesterolemia. 

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