Chapter·BiochemistryGlycogen storage diseases

GSD type VI (Hers disease)Downloads

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1

A 67-year-old man with hypertension comes to the emergency department because of progressively worsening abdominal pain that started 1 week ago. The pain is localized to the right upper quadrant. He has also noticed yellowing of his eyes and skin during this time period. Physical examination shows jaundice, a distended abdomen, and tender hepatomegaly. There is no jugular venous distention. Laboratory studies show a hemoglobin concentration of 19.2 g/dL, aspartate aminotransferase of 420 U/L, alanine aminotransferase of 318 U/L, and total bilirubin of 2.2 mg/dL. Which of the following is the most likely cause of this patient's symptoms?

AThickened pericardium

BHepatic steatosis

CHepatotropic viral infection

DIncreased iron absorption

EHepatic vein obstruction

2

A 3-month-old girl is brought to the emergency department by her parents after she appeared to have a seizure at home. On presentation, she no longer has convulsions though she is still noted to be lethargic. She was born through uncomplicated vaginal delivery and was not noted to have any abnormalities at the time of birth. Since then, she has been noted by her pediatrician to be falling behind in height and weight compared to similarly aged infants. Physical exam reveals an enlarged liver, and laboratory tests reveal a glucose of 38 mg/dL. Advanced testing shows that a storage molecule present in the cells of this patient has abnormally short outer chains. Which of the following enzymes is most likely defective in this patient?

ADebranching enzyme

BHepatic phosphorylase

CGlucose-6-phosphatase

DMuscle phosphorylase

EBranching enzyme

3

A 10-year-old boy is brought to the emergency department due to vomiting and weakness. He is attending a summer camp and was on a hike with the other kids and a camp counselor. His friends say that the boy skipped breakfast, and the counselor says he forgot to pack snacks for the kids during the hike. The child’s parents are contacted and report that the child has been completely healthy since birth. They also say there is an uncle who would have to eat regularly or he would have similar symptoms. At the hospital, his heart rate is 90/min, respiratory rate is 17/min, blood pressure is 110/65 mm Hg, and temperature is 37.0°C (98.6°F). Physical examination reveals a visibly lethargic child with slight disorientation to time and place. Mild hepatosplenomegaly is observed but no signs of dehydration are noted. A blood sample is drawn, and fluids are started via an intravenous line. Lab report Serum glucose 44 mg/dL Serum ketones absent Serum creatinine 1.0 mg/dL Blood urea nitrogen 32 mg/dL Alanine aminotransferase (ALT) 425 U/L Aspartate aminotransferase (AST) 372 U/L Hemoglobin (Hb%) 12.5 g/dL Mean corpuscular volume (MCV) 80 fl Reticulocyte count 1% Erythrocyte count 5.1 million/mm3 Which of the following is most likely deficient in this patient?

AAcyl-CoA dehydrogenase

Bα-glucosidase

CGlucose-6-phosphatase

DAcetyl-CoA carboxylase

ENicotinic acid

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