A 5-month-old infant presented with symmetrical profound muscle weakness, tachypnea, tachycardia, and poor feeding. His chest x-ray showed cardiomegaly and his ECG revealed short PR interval. Of the following, the MOST likely glycogen storage disease is

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Endocrine System Pediatrics Questions

Question 1 of 5

A 5-month-old infant presented with symmetrical profound muscle weakness, tachypnea, tachycardia, and poor feeding. His chest x-ray showed cardiomegaly and his ECG revealed short PR interval. Of the following, the MOST likely glycogen storage disease is

Correct Answer: C

Rationale: Type II (Pompe) causes profound muscle weakness, cardiomyopathy (cardiomegaly), and respiratory issues due to glycogen accumulation in muscles and heart, with short PR interval on ECG, matching this presentation.

Question 2 of 5

Sudden infant death syndrome is reported in infants with

Correct Answer: C

Rationale: Medium-chain acyl-CoA dehydrogenase (MCAD) deficiency is a known cause of SIDS due to hypoketotic hypoglycemia during fasting stress, more commonly than other fatty acid oxidation disorders.

Question 3 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 4 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 5 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.

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