The lipid storage diseases are a group of disorders that result from specific enzymatic deficiencies. Each statement about these disorders is true EXCEPT

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Pediatric Endocrine Nursing Questions Questions

Question 1 of 5

The lipid storage diseases are a group of disorders that result from specific enzymatic deficiencies. Each statement about these disorders is true EXCEPT

Correct Answer: B

Rationale: Diagnosis typically uses leukocyte enzyme assays or genetic testing, not liver biopsy, which is invasive and less common.

Question 2 of 5

Matching: Mutation in KIT gene

Correct Answer: D

Rationale: Piebaldism results from KIT gene mutations, causing patchy depigmentation.

Question 3 of 5

To evaluate the patient in the previous question, all of the following are appropriate EXCEPT

Correct Answer: D

Rationale: 17-hydroxyprogesterone is for congenital adrenal hyperplasia, not organic acidemia evaluation.

Question 4 of 5

Matching: 3-Methylglutaconic aciduria type II

Correct Answer: D

Rationale: Type II (Barth syndrome) is X-linked with cardiomyopathy.

Question 5 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.

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