Galactosemia is an autosomal recessive disease characterized by all the following EXCEPT

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Pediatric Endocrine System Questions

Question 1 of 5

Galactosemia is an autosomal recessive disease characterized by all the following EXCEPT

Correct Answer: C

Rationale: Galactosemia (galactose-1-phosphate uridyltransferase deficiency) causes hyperbilirubinemia, coagulopathy, glycosuria, and aminoaciduria from galactose accumulation, but hyperglycemia is not typical as glucose metabolism is unaffected.

Question 2 of 5

A relative parents brought their underweight 6-month-old female infant who has high foreheads, flat orbital ridges, widely open fontanelles, hepatomegaly, hypotonia, and nystagmus; she had history of recurrent seizures. Of the following, the MOST likely diagnosis is

Correct Answer: B

Rationale: Zellweger syndrome, a peroxisomal disorder, presents with dysmorphic features (high forehead, flat ridges), hepatomegaly, hypotonia, seizures, and nystagmus, consistent with this case and consanguinity.

Question 3 of 5

In children with Down syndrome (DS), the type of leukemia varies by age. In those younger than 2 years, it is generally

Correct Answer: D

Rationale: In children with DS younger than 2 years, the type is generally acute megakaryoblastic leukemia; in individuals older than 3 years, acute lymphoblastic leukemia predominates.

Question 4 of 5

Matching: For each inborn error of amino acid metabolism, select the correct urine odor - Glutaric acidemia (type II)

Correct Answer: C

Rationale: Glutaric acidemia type II produces a sweaty feet-like odor due to accumulation of organic acids from fatty acid oxidation defects.

Question 5 of 5

Matching: Match the disease with its enzyme - Iduronidase

Correct Answer: A

Rationale: Hurler syndrome (MPS I) results from iduronidase deficiency, causing mucopolysaccharide buildup.

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