The mucolipidoses result from the abnormal targeting of lysosomal enzymes to the lysosome. Each statement about the mucolipidoses is correct EXCEPT

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Endocrine System Pediatrics Questions

Question 1 of 5

The mucolipidoses result from the abnormal targeting of lysosomal enzymes to the lysosome. Each statement about the mucolipidoses is correct EXCEPT

Correct Answer: D

Rationale: Not all mucolipidoses (e.g., type III) lead to early childhood death; severity varies.

Question 2 of 5

In general, albinism is characterized by all of the following EXCEPT

Correct Answer: D

Rationale: Nail hypoplasia is not a feature of albinism; others relate to melanin deficiency.

Question 3 of 5

A 4-year-old blue-eyed white female manifests a malar flush, mild mental retardation, subluxation of the ocular lens, iridodonesis, and Marfanoid-like features (tall, thin, arachnodactyly). The most likely diagnosis is

Correct Answer: D

Rationale: Homocystinuria mimics Marfan syndrome with lens dislocation and retardation due to homocysteine accumulation.

Question 4 of 5

Matching: Isovaleric acidemia

Correct Answer: D

Rationale: Isovaleric acidemia can present with pancytopenia during crises due to bone marrow suppression.

Question 5 of 5

In addition to routine supportive care, what additional therapy would be most appropriate for the child in the previous question?

Correct Answer: A

Rationale: Vitamin B12 treats methylmalonic acidemia in B12-responsive forms, reducing toxic metabolites.

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