Matching: Match the disease with its enzyme - β-Hexosaminidase A

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Endocrine System Pediatric Questions

Question 1 of 5

Matching: Match the disease with its enzyme - β-Hexosaminidase A

Correct Answer: C

Rationale: Tay-Sachs disease is caused by β-hexosaminidase A deficiency, resulting in GM2 ganglioside buildup.

Question 2 of 5

A 10-year-old girl is noted on a routine physical examination to have splenomegaly. Laboratory testing reveals thrombocytopenia and moderate edema, and she is referred to a hematologist for further evaluation. Bone marrow aspiration is performed and reveals the presence of Gaucher cells. When discussing therapeutic options with the parents of this child, it would be most appropriate to include which of the following statements?

Correct Answer: D

Rationale: Enzyme replacement therapy (e.g., imiglucerase) effectively reverses hematologic issues in Gaucher type I.

Question 3 of 5

The best approach to the diagnosis of the patient in the previous question is

Correct Answer: B

Rationale: Fumarylacetoacetate hydrolase assay confirms tyrosinemia type I by detecting enzyme deficiency.

Question 4 of 5

Matching: Hemizygotic male has complete syndrome

Correct Answer: D

Rationale: Nettleship-Falls ocular albinism is X-linked, fully expressed in hemizygous males.

Question 5 of 5

A 1-week-old infant presents with poor feeding, vomiting, lethargy, and opisthotonus posturing. The critical impression is meningitis, but all cultures are negative. Seizures occur and hypoglycemia is documented; however, the seizures do not stop after the hypoglycemia is corrected with intravenous glucose. The arterial blood gas is 7.10 PCO2 23, PO2 90, with an anion gap of 35. A CT scan demonstrates cerebral edema. The most likely etiology is

Correct Answer: D

Rationale: Organic acidemia (e.g., propionic or methylmalonic) causes acidosis, anion gap, seizures, and cerebral edema unresponsive to glucose.

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