A 27-day-old girl presented with abnormal bicycling movement, lethargy, poor feeding, and repeated vomiting for the last 7 days, and then she developed rapid breathing, opisthotonos, and hypertonicity with bad odor urine. Of the following, the MOST effective mode of therapy is

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Endocrine System Pediatrics Questions

Question 1 of 5

A 27-day-old girl presented with abnormal bicycling movement, lethargy, poor feeding, and repeated vomiting for the last 7 days, and then she developed rapid breathing, opisthotonos, and hypertonicity with bad odor urine. Of the following, the MOST effective mode of therapy is

Correct Answer: D

Rationale: MSUD, indicated by symptoms and urine odor, is best managed with a diet low in branched-chain amino acids to reduce toxic metabolites, more effective than hydration (A), dialysis (B), transplantation (C), or calories alone (D).

Question 2 of 5

All the following lipidoses are inherited as an autosomal recessive traits EXCEPT

Correct Answer: A

Rationale: Fabry disease is X-linked, unlike the autosomal recessive Krabbe (B), Gaucher (C), Tay-Sachs (D), and Niemann-Pick (E).

Question 3 of 5

The definitive diagnosis of type I glycogen storage disease GSD is by

Correct Answer: B

Rationale: Liver biopsy showing glycogen accumulation and enzyme deficiency (glucose-6-phosphatase) definitively diagnoses GSD I, more specific than EMG (A), muscle biopsy (C), gene analysis (D), or fibroblasts (E).

Question 4 of 5

A 20-day-old girl presented with prolonged neonatal jaundice, lethargy, and poor feeding treated as sepsis by empirical antibiotics and nothing by mouth. The patient got partial response, when oral feeding resumed, the condition deteriorate again. In this condition, the blood culture MOST likely reveals growth of

Correct Answer: A

Rationale: Galactosemia increases E. coli sepsis risk from galactose-induced immune suppression, exacerbated by feeding resumption, more common than other pathogens (B-E) in this context.

Question 5 of 5

A 9-year-old boy presented with tiredness on exertion; cardiac auscultation reveals a murmur in aortic area; echo study showed aortic valve disease. On examination he had mild coarse facial features, corneal clouding, joint stiffness, and mild dysostosis multiplex but normal intelligence and stature. Of the following, the MOST likely diagnosis is

Correct Answer: B

Rationale: Scheie disease (mild MPS I) fits with normal intelligence, stature, corneal clouding, and cardiac issues, unlike severe Hurler (A), X-linked Hunter (C), skeletal Morquio (D), or CNS Sanfilippo (E).

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