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Topics in Genetic Counseling - Lysosomal Storage Diseases - I

Total questions: 12

Worksheet time: 6mins

Name
Class
Date
1.

What is the main function of the lysosome inside of the cell?

a)

cell structure that produces ATP through oxidative phosphorylation

b)

cell structure that has enzymes that breakdown macromolecules

c)

cell structure that processes proteins for transport within the cell

d)

cell structure responsible for producing proteins from mRNA

2.

What is the collective incidence of lysosomal storage diseases?

a)

1:70,000 - 1:100,000

b)

1:7000 - 1:10,000

c)

1:700 - 1:1000

d)

1:700,000-1,000,000

e)

Incidence is not able to be calculated due to rareness

3.

What is the most common LSD?

a)

Gaucher disease

b)

Fabry disease

c)

Krabbe disease

d)

MPSI

e)

MPSII

4.

Most of the LSD's are autosomal recessive but which LSD's are X-linked?

a)

MPSI, MPSII and MPSIV

b)

MPSI, Hunter and Gaucher

c)

Fabry, Danon (dom) and Hunter

d)

Pompe, Gaucher and Fabry

e)

Hunter, Danon (dom) and MPSIV

5.

More than half of the LSD's are associated with...

a)

Failure to thrive

b)

Craniofacial malformations

c)

Cardiac malfromations

d)

CNS involvement

e)

Hypotonia

6.

The most common type of Gaucher diseease is...

a)

Type 1

b)

Type 2

c)

Type 3

d)

Perinatal-lethal

e)

Cardiovascular

7.

If you are thinking of ordering testing for Gaucher disease, one concern that you might have about the selected testing is...

a)

high de novo frequency

b)

mostly del/dup variants

c)

presence of a pseudogene

d)

multiple functional spliced variants

e)

gene is near a chromosomal breakpoint

8.

The most severe type of Gaucher disease is...

a)

Type 1

b)

Type 2

c)

Type 3

d)

Perinatal-lethal

e)

Cardiovascular

9.

For Fabry disease, which set of facts are true?

a)

X-linked, adult onset

b)

X-linked, childhood onset

c)

Autosomal recessive, adult onset,

d)

Autosomal recessive, childhood onset

e)

Autosomal dominant, childhood onset

10.

Some patients with Fabry disease may be considered pain-drug seeking because of what symptom?

a)

Recurrent fever

b)

Corneal whorls

c)

Heat/cold intolerance

d)

Acroparesthesia

e)

Left ventricular myopathy

11.

With regard to Pompe disease, which set of facts are true?

a)

Autosomal dominant, encoded by GAA gene, usually fatal

b)

Autosomal dominant, encoded by GBA gene, not fatal

c)

Autosomal recessive, encoded by GAA gene, usually fatal

d)

Autosomal recessive, encoded by GBA gene, not fatal

12.

The hallmark symptom of infantile onset Pompe disease would be...

a)

failure to thrive

b)

sleep distrubances

c)

hypotonia

d)

cardiomegaly/cardiomyopathy

e)

gait abnormalities