Rare Diseases and Pipeline


HoFH (homozygous familial hypercholesterolemia) is an uncommon genetic condition that is inherited from both parents and leads to high levels of cholesterol in your blood.  People with HoFH have blood cholesterol levels that are exceptionally high, usually between 10 and 20 mmol/L and possibly even higher.  At these levels, the cholesterol accumulates in the arteries much faster than in the normal population leading to premature heart disease.  People with HoFH do not always respond to usual lipid lowering drugs such as statins and so need other therapies to lower their cholesterol levels.

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