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Candidate drug for familial hypercholesterolaemia needs more work

BMJ 2007; 334 doi: (Published 18 January 2007) Cite this as: BMJ 2007;334:118

People with severe familial hypercholesterolaemia have non-functioning receptors for low density lipoprotein (LDL) cholesterol. This defect leads to sky high serum concentrations of total and LDL cholesterol, early cardiovascular disease, and death. Treatment options are limited because conventional drug treatments don't work, …

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