他汀类药物、依折麦布和PCSK9抑制剂的出现极大改善了家族性高胆固醇血症(FH)的预后。然而,尽管进行了最大限度的降脂治疗,许多FH患者的低密度脂蛋白胆固醇(LDL-C)水平仍达不到指南推荐的水平。不依赖于低密度脂蛋白(LDL)受体活性而降低LDL的 ...
Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment.