Type III Glycogen Storage Disease Treatment & Management
- Author: Wayne E Anderson, DO, FAHS, FAAN; Chief Editor: George T Griffing, MD more...
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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. 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.
- 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.
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.
See the list below:
- 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.
Cornstarch therapy may be beneficial in reducing hypoglycemia.
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