A constant threat of homocystinuria is

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Endocrine System Pediatrics Questions

Question 1 of 5

A constant threat of homocystinuria is

Correct Answer: D

Rationale: Homocystinuria’s constant threat is arterial thromboses due to homocysteine’s vascular toxicity, a life-threatening complication outweighing skeletal (B, C), ocular (A), or cognitive (D) issues.

Question 2 of 5

Lipidoses affect many solid organs, cherry-red spots is seen in

Correct Answer: D

Rationale: GM2 gangliosidosis (Tay-Sachs) classically features cherry-red spots in the macula from lipid accumulation in retinal ganglion cells, a hallmark finding.

Question 3 of 5

The condition with the LEAST association with chromosome 22q deletion is

Correct Answer: D

Rationale: All except E (Williams syndrome, linked to 7q deletion) represent a continuum of findings due to chromosome 22q deletion, including cleft palate, cardiac defects, and T-cell dysfunction.

Question 4 of 5

Matching: Match the disease with its enzyme - α-Galactosidase

Correct Answer: D

Rationale: Fabry disease is caused by α-galactosidase deficiency, leading to globotriaosylceramide accumulation.

Question 5 of 5

A 13-month-old infant is found comatose in bed after sleeping later than usual. On physical examination, the infant is afebrile and of normal size, and the liver is palpated 4 cm below the costal margin. The plasma glucose level is 15 mg/dL, the bicarbonate level is 20 mEq/L, BUN is 35 mg/dL, ammonia is 295 μmol/L, AST is 320 U/L, ALT is 425 U/L, and bilirubin is normal. Urinalysis is negative for glucose, ketones, protein, and reducing substances. Which of the following is the most likely diagnosis?

Correct Answer: B

Rationale: Type I glycogen storage disease (von Gierke) causes severe hypoglycemia, hepatomegaly, and elevated ammonia/liver enzymes due to glucose-6-phosphatase deficiency.

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