A 52-year-old woman presents with xanthomas on her eyelids and Achilles tendons, along with premature coronary artery disease. Laboratory studies show LDL cholesterol of 420 mg/dL despite atorvastatin 80 mg daily, HDL of 35 mg/dL, and normal triglycerides. Her father and paternal grandfather both had myocardial infarctions in their 40s. Which of the following is the most likely genetic defect?

  1. A)Loss-of-function mutation in apolipoprotein B-100
  2. B)Gain-of-function mutation in PCSK9
  3. C)Loss-of-function mutation in the LDL receptor geneGABARITO
  4. D)Loss-of-function mutation in lipoprotein lipase
  5. E)Gain-of-function mutation in HMG-CoA reductase

Explicação

Familial hypercholesterolemia due to LDL receptor deficiency causes severe LDL elevation (often >300 mg/dL), tendon xanthomas, and premature CAD. Heterozygous mutations affect ~1 in 500 people; homozygous mutations cause even more severe disease. Statins have ... Ver explicação completa e trilha adaptativa →

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