Serum amino acids are normal and the urine orotic acid is elevated in the patient in the previous question. The most likely diagnosis is

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Pediatric Endocrine Nursing Questions Questions

Question 1 of 5

Serum amino acids are normal and the urine orotic acid is elevated in the patient in the previous question. The most likely diagnosis is

Correct Answer: D

Rationale: Elevated orotic acid with hyperammonemia points to ornithine transcarbamylase (OTC) deficiency.

Question 2 of 5

A 7-year-old male previously 'A' student demonstrates progressive hyperactivity is diagnosed with attention deficit hyperactivity disorder. Despite therapy, his school performance deteriorates. He has difficulty understanding people when spoken to on the telephone. Seizures developed 6 months later. Physical examination reveals slight diffuse hyperpigmentation, spasticity, and reduced deep tendon reflexes. The most likely diagnosis is

Correct Answer: D

Rationale: X-linked adrenoleukodystrophy (ALD) causes progressive neurologic decline, seizures, and adrenal insufficiency (pigmentation).

Question 3 of 5

Matching: Lipidosis - Sandhoff

Correct Answer: C

Rationale: Sandhoff disease has both cherry red spot and hepatosplenomegaly.

Question 4 of 5

Treatment of the patient in the previous two questions is best achieved with

Correct Answer: B

Rationale: Enzyme replacement with β-glucosidase (e.g., imiglucerase) treats Gaucher disease.

Question 5 of 5

Matching: Glycogen storage diseases - Anderson

Correct Answer: D

Rationale: Anderson (type IV) causes cirrhosis and failure to thrive.

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