Matching: Match the disease with its enzyme - Iduronidase

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Pediatric Endocrine System Questions

Question 1 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.

Question 2 of 5

The parents of a 2-year-old boy with Tay-Sachs disease ask you about the availability of prenatal testing in their pregnancy. When counseling this couple about prenatal diagnosis, it would be most appropriate to include which one of the following statements:

Correct Answer: A

Rationale: Prenatal testing for Tay-Sachs via CVS at 10 weeks is standard and reliable, given its autosomal recessive inheritance (25% risk per pregnancy).

Question 3 of 5

Dietary restriction of phenylalanine during pregnancy to serum levels of 10 mg/dL prevents all of the following EXCEPT

Correct Answer: B

Rationale: Phenylalanine restriction prevents fetal damage (microcephaly, heart defects, retardation) but not maternal preeclampsia, a pregnancy-specific condition.

Question 4 of 5

Matching: Platelet dysfunction

Correct Answer: B

Rationale: Hermansky-Pudlak syndrome includes platelet dysfunction and bleeding tendencies.

Question 5 of 5

Additional complications that may occur in the patient in the previous question include all of the following EXCEPT

Correct Answer: D

Rationale: Cardiomyopathy is not typical in homocystinuria; others (thrombosis, osteoporosis) are common.

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