Learn About Neimenn-Pick Disease Based On MCQs (20MCQs)

Here are MCQs on Niemann-Pick disease to help you clear your basics:

1) What is the primary cause of Niemann-Pick disease?

A) Deficiency of acid sphingomyelinase

B) Deficiency of glucocerebrosidase

C) Deficiency of hexosaminidase A

D) Deficiency of galactocerebrosidase

Ans - A

2) Which of the following is a characteristic feature of Niemann-Pick disease?

A) Accumulation of glucocerebroside in cells

B) Accumulation of sphingomyelin in cells

C) Accumulation of gangliosides in cells

D) Accumulation of cholesterol in cells

Ans - B

3) What is the primary organ affected in Niemann-Pick disease?

A) Brain

B) Liver

C) Spleen

D) All of the above

Ans - D

4) Which of the following is a common symptom of Niemann-Pick disease?

A) Seizures

B) Ataxia

C) Dementia

D) All of the above

Ans - D

5) What is the mode of inheritance of Niemann-Pick disease?

A) Autosomal dominant

B) Autosomal recessive

C) X-linked dominant

D) X-linked recessive

Ans - B

6) Which of the following is used for the diagnosis of Niemann-Pick disease?

A) Enzyme assay

B) Genetic testing

C) Imaging studies

D) All of the above

Ans - D

7) What is the treatment for Niemann-Pick disease?

A) Enzyme replacement therapy

B) Bone marrow transplantation

C) Gene therapy

D) Supportive care

Ans - D

8) Which of the following is a complication of Niemann-Pick disease?

A) Respiratory failure

B) Cardiac failure

C) Hepatic failure

D) All of the above

Ans - D

9) What is the prognosis for patients with Niemann-Pick disease?

A) Good

B) Fair

C) Poor

D) Variable

Ans - C

10) Which of the following is a type of Niemann-Pick disease?

A) Type A

B) Type B

C) Type C

D) All of the above

Ans - D

11) What is the age of onset for Niemann-Pick disease Type A?

A) Infancy

B) Childhood

C) Adolescence

D) Adulthood

Ans - A

12) Which of the following is a characteristic feature of Niemann-Pick disease Type C?

A) Accumulation of sphingomyelin in cells

B) Accumulation of cholesterol in cells

C) Deficiency of acid sphingomyelinase

D) Deficiency of NPC1 protein

Ans - D

13) What is the primary cause of death in patients with Niemann-Pick disease?

A) Respiratory failure

B) Cardiac failure

C) Hepatic failure

D) Neurological deterioration

Ans - D

14) Which of the following is a potential therapeutic approach for Niemann-Pick disease?

A) Gene therapy

B) Enzyme replacement therapy

C) Stem cell transplantation

D) All of the above

Ans - D

15) What is the current status of treatment for Niemann-Pick disease?

A) Curative

B) Symptomatic

C) Experimental

D) Supportive

Ans - D

16) What is the role of NPC1 protein in Niemann-Pick disease Type C?

A) To transport cholesterol out of lysosomes

B) To transport sphingomyelin out of lysosomes

C) To regulate acid sphingomyelinase activity

D) To inhibit apoptosis

Ans - A

17) According to recent studies, what is the potential therapeutic target for Niemann-Pick disease?

A) Acid sphingomyelinase

B) NPC1 protein

C) Histone deacetylase

D) Autophagy-related proteins

Ans - D

18) What is the result of recent clinical trials using miglustat for the treatment of Niemann-Pick disease Type C?

A) Significant improvement in cognitive function

B) Stabilization of neurological symptoms

C) Reduction in liver and spleen size

D) No significant clinical benefit

Ans - B

19) According to recent research, what is the link between Niemann-Pick disease and Alzheimer's disease?

A) Both diseases involve the accumulation of beta-amyloid peptides

B) Both diseases involve the dysfunction of lysosomes

C) Both diseases involve the impairment of autophagy

D) Both diseases involve the activation of inflammatory pathways

Ans - B

20) What is the current direction of research for the treatment of Niemann-Pick disease?

A) Enzyme replacement therapy

B) Gene therapy

C) Small molecule therapies targeting specific disease mechanisms

D) Stem cell transplantation

Ans - C