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FH
- Pathology. familial hypercholesterolemia.
Example Sentences
People with FH must take daily statins and other drugs to control their cholesterol levels, but many struggle to keep to the life-long regimen.
FH patients still make some LDL receptors, and Verve’s strategy is to keep those molecules around for longer by altering the liver’s gene for PCSK9, an enzyme that normally removes the receptors from cells.
Endocrinologist Anne Goldberg of Washington University in St. Louis, who treats FH patients, says that although the Verve treatment “could be a game-changer,” she wants to see more safety data.
Verve plans to test its treatment in a total of about 40 FH patients.
He says the PCSK9 base editor could one day be used to treat people who don’t have FH but have early heart disease.
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