All the following are recognized feature of Niemann-pick disease type B EXCEPT

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Pediatric Endocrine Exam Questions Questions

Question 1 of 5

All the following are recognized feature of Niemann-pick disease type B EXCEPT

Correct Answer: C

Rationale: Niemann-Pick type B features normal IQ (A), lung disease (B), minimal neurology (D), and occasional cherry-red spots (E). Prolonged jaundice (C) is more typical of type A or other conditions.

Question 2 of 5

Irreversible complication of untreated galactosemia is

Correct Answer: D

Rationale: Untreated galactosemia causes irreversible intellectual disability from brain damage, more permanent than cataracts (B), sepsis (A), or organ injuries (C, D), which may be treatable.

Question 3 of 5

Enzyme replacement is the main therapy of Hurler disease, it improve all the following EXCEPT

Correct Answer: D

Rationale: ERT in Hurler syndrome (MPS I) improves growth (A), mobility (B), organomegaly (C), and apnea (E), but cognitive impairment (D) persists due to CNS GAG accumulation.

Question 4 of 5

In mucopolysaccharidosis, disproportionate severe CNS involvement with mild physical features is unique to

Correct Answer: D

Rationale: Sanfilippo disease (MPS III) uniquely features severe CNS involvement (dementia) with milder somatic findings, unlike others (A-D).

Question 5 of 5

All the following drugs are unsafe in acute intermittent porphyria EXCEPT

Correct Answer: A

Rationale: Nifedipine is safe in AIP, unlike diclofenac (B), cimetidine (C), valproic acid (D), and carbamazepine (E), which induce heme synthesis and exacerbate attacks.

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