Type III Glycogen Storage Disease Treatment & Management

Updated: May 25, 2017
  • Author: Wayne E Anderson, DO, FAHS, FAAN; Chief Editor: George T Griffing, MD  more...
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Treatment

Medical Care

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  • In general, no specific treatment exists for GSDs.

  • In some cases, diet therapy is helpful. Meticulous adherence to a dietary regimen may reduce liver size, prevent hypoglycemia, allow for reduction in symptoms, and allow for growth and development.

  • Zingone and colleagues demonstrated the abolition of the murine clinical manifestations of Von Gierke disease with a recombinant adenoviral vector. [12] These findings suggest that corrective gene therapy for GSDs may be possible in humans.

  • An encouraging study by Bijvoet and colleagues provides evidence of successful enzyme replacement for the mouse model of Pompe disease, which may lead to therapies for other enzyme deficiencies. [13]

  • Valayannopoulos et al reported positive treatment of an infant with GSD III complicated by severe cardiomyopathy using synthetic ketone bodies, along with a low-carbohydrate, high-lipid, and high-protein diet. [14]

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Surgical Care

Liver transplantation may be indicated for patients with hepatic malignancy. Whether transplantation prevents further complications is not clear, although a study by Matern and colleagues demonstrated posttransplantation correction of metabolic abnormalities. [15]

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Consultations

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  • Consultation with a neurologist with special training in muscle physiology may help in establishing the diagnosis.

  • A consultation with a hepatologist-liver specialist may be helpful in the evaluation of liver abnormalities.

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Diet

Cornstarch therapy may be beneficial in reducing hypoglycemia.

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