Plasma very-long-chain fatty acids are elevated in all of the following peroxisomal disorders EXCEPT

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Endocrine System Pediatric Questions

Question 1 of 5

Plasma very-long-chain fatty acids are elevated in all of the following peroxisomal disorders EXCEPT

Correct Answer: B

Rationale: Rhizomelic chondrodysplasia punctata involves plasmalogen synthesis defects, not VLCFA elevation, unlike the others.

Question 2 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 3 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 4 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.

Question 5 of 5

Matching: Dietary biotin deficiency

Correct Answer: C

Rationale: Dietary biotin deficiency, often from raw egg consumption (avidin binds biotin), causes skin and hair issues.

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