Transient tyrosinemia of the newborn responds to treatment with

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Pediatric Endocrine Exam Questions Questions

Question 1 of 5

Transient tyrosinemia of the newborn responds to treatment with

Correct Answer: A

Rationale: Transient tyrosinemia of the newborn, due to immature liver enzymes, resolves with ascorbic acid (vitamin C), which enhances tyrosine metabolism, unlike other vitamins or formulas.

Question 2 of 5

Lipidoses affect many solid organs, optic atrophy is seen in

Correct Answer: D

Rationale: Galactosylceramide lipidosis (Krabbe) causes optic atrophy due to white matter degeneration, unlike other listed lipidoses where it’s less prominent.

Question 3 of 5

Williams syndrome is characterized by

Correct Answer: C

Rationale: Williams syndrome results from a small deletion on chromosome 7q, causing moderate intellectual disability (IQ 50-60), a 'cocktail party' personality, and occasional musical ability, with about 10% showing autism features.

Question 4 of 5

A previously healthy 4-month-old now manifests increasing hypotonia and poor feeding. Physical examination reveals macroglossia, a gallop rhythm and tachycardia, marked flaccidity, but normal mental status. Laboratory studies reveal a blood glucose level of 85 mg/dL and sinus tachycardia with a shortened PR interval on an electrocardiogram. The most helpful diagnostic study would be

Correct Answer: C

Rationale: Pompe disease (glycogen storage disease type II) matches these symptoms (cardiomyopathy, hypotonia, macroglossia); muscle biopsy confirms glycogen accumulation.

Question 5 of 5

A 15-year-old Ashkenazi Jewish girl is seen because of chronic fatigue. On examination, she seems pale and thin and has a somewhat large abdomen. Her spleen is felt in the iliac fossa. She is mentally alert and has a history of normal development and normal school performance. Her blood count shows hemoglobin of 9.0 g/dL and a white blood cell count of 3000 with normal differential and no abnormal cells. Platelet count is 60,000. The likely diagnosis is

Correct Answer: C

Rationale: Gaucher type I, common in Ashkenazi Jews, presents with splenomegaly, anemia, and thrombocytopenia without neurologic involvement.

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