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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Glycogen storage disease type 4


Other Names for this Disease
  • Amylopectinosis
  • Andersen disease
  • Brancher deficiency
  • Cirrhosis, familial, with deposition of abnormal glycogen
  • Glycogen branching enzyme deficiency
More Names
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Treatment


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How might glycogen storage disease type 4 be treated?

Management of glycogen storage disease type 4 typically focuses on the signs and symptoms that are present in each individual. Studies have show that in some cases, strict dietary therapy can help to maintain normal levels of glucose in the blood, reduce liver size, reduce symptoms, and allow for improved growth and development. Growing evidence indicates that a high-protein diet may improve muscle function in individuals with weakness or exercise intolerance and slow disease progression. Supportive care is typically needed for complications such as liver failure, heart failure, and neurologic dysfunction. Liver transplantation may be necessary for individuals with progressive liver disease. Individuals with cardiomyopathy may require the use of certain medications.[1][2]
Last updated: 9/8/2011

References
  1. Wayne E Anderson. Glycogen Storage Disease, Type IV Treatment & Management. eMedicine. November 12, 2009; http://emedicine.medscape.com/article/119690-treatment#showall. Accessed 9/8/2011.
  2. Marsden D. Andersen Disease (GSD IV). NORD. 2012; http://www.rarediseases.org/rare-disease-information/rare-diseases/byID/394/viewAbstract. Accessed 12/23/2012.


Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Glycogen storage disease type 4. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.