The prognosis of glycogen storage diseases is good in

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Pediatric Endocrine Exam Questions Questions

Question 1 of 5

The prognosis of glycogen storage diseases is good in

Correct Answer: D

Rationale: Type V (McArdle) has a good prognosis as it primarily causes exercise intolerance without systemic failure. Type 1a (A) and 1b (B) require careful management, type II (C) is fatal without treatment, and type IV (D) progresses to liver failure.

Question 2 of 5

Sweaty feet odor is seen in untreated infants with

Correct Answer: A

Rationale: Isovaleric acidemia produces a distinctive 'sweaty feet' odor from isovaleryl-CoA accumulation, unlike other listed disorders.

Question 3 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 4 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 5 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.

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