PIK3C2A and familial hypercholesterolemia: The most common TRAEs (≥10%) were AST elevated (30%), ALT elevated (29%), nausea (28%), hypercholesterolemia (27%), vomiting (24%), hypertriglyceridemia (21%), hypertension (17%), CPK elevated (16%), diarrhea (15%), rash (11%), γ-GGT elevated (10%), and hyperlipidemia (10%).