Advanced Rare Genetic Disorder Challenge

A rigorous assessment of obscure inherited diseases designed for seasoned medical professionals.

medical geneticsrare diseasesgeneticspathophysiologydiagnosishereditary disordersclinical reasoning
Difficulty:HARD

Quiz Details

Questions7
CategoryHealth & Medicine
DifficultyHARD
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Quiz Questions

Answer all questions below and test your knowledge.

  1. 1

    Which enzymatic defect underlies Fabry disease?

    Question 1
  2. 2

    A newborn presents with profound hypotonia, seizures, and elevated very long‑chain fatty acids. Which peroxisomal disorder is most likely?

    Question 2
  3. 3

    Which mutation type is most frequently associated with Marfan syndrome?

    Question 3
  4. 4

    In hereditary hemorrhagic telangiectasia, what vascular anomaly characterizes the pulmonary circulation?

    Question 4
  5. 5

    Which therapy has shown disease‑modifying benefit in early‑onset Huntington disease?

    Question 5
  6. 6

    A 25‑year‑old woman with a family history of early‑onset breast cancer tests positive for a BRCA1 truncating variant. What surveillance protocol is recommended after age 30?

    Question 6
  7. 7

    Which cytokine is most implicated in the pathogenesis of familial hemophagocytic lymphohistiocytosis type 2?

    Question 7

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