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Diseases

Genetic and Rare Diseases Information Center (GARD)

Danon disease


Other Names for this Disease
  • Vacuolar cardiomyopathy and myopathy X-linked
  • X-linked vacuolar cardiomyopathy and myopathy
  • Antopol disease
  • Pseudoglycogenosis 2
  • Glycogen storage disease limited to the heart
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

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How might Danon disease be treated?

Treatment is aimed at addressing the symptoms present in each individual. This may require a team of specialists in addition to the primary care physician, including a cardiologist, neurologist, ophthalmologist, geneticist, genetic counselor, rehabilitation physician, educational specialist, and physical therapist. Because Danon disease can be associated with rapidly progressive cardiomyopathy and sudden death, careful monitoring of heart disease is required. Medications for heart disease may be a first line of treatment. Aggressive interventions may be recommended for people showing signs of progressive heart failure (e.g., early intervention with heart transplantation or implantable cardioverter-defibrillator). Assessment of muscle strength should be performed regularly. Physical therapy may help maintain muscle strength and flexibility. Males with intellectual disabilities should receive appropriate educational interventions.[1]  
Last updated: 7/12/2016

References
  1. Taylor MRG, D'souza R. Danon disease. National Organization for Rare Disorders (NORD). 2015; http://rarediseases.org/rare-diseases/danon-disease/.


Clinical Trials & Research for this Disease

  • Orphanet lists European clinical trials, research studies, and patient registries enrolling people with this condition. 
Other Names for this Disease
  • Vacuolar cardiomyopathy and myopathy X-linked
  • X-linked vacuolar cardiomyopathy and myopathy
  • Antopol disease
  • Pseudoglycogenosis 2
  • Glycogen storage disease limited to the heart
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.