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

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Endocrine System Pediatrics Questions

Question 1 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 2 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).

Question 3 of 5

All the following are skeletal manifestations of Morquio disease EXCEPT

Correct Answer: D

Rationale: Morquio disease (MPS IV) causes kyphosis (A), genua valga (B), waddling gait (C), and short trunk/neck (D). Bullet-shaped phalanges (E) are typical of Hurler, not Morquio.

Question 4 of 5

All the following are clinical features of acute intermittent porphyria EXCEPT

Correct Answer: D

Rationale: AIP features neuropathy (A), tachycardia (B), abdominal pain (C), and bladder issues (D). Photosensitivity (E) is absent, typical of cutaneous porphyrias.

Question 5 of 5

The least well-defined long-term sequelae of severe prolonged hypoglycemia is

Correct Answer: B

Rationale: Personality affection (B) is less well-defined as a hypoglycemia sequela compared to cerebral palsy (A), cognitive impairment (C), seizures (D), or autonomic issues (E), which are better documented.

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