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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

Newline Maker

How might the cardiomyopathy in Danon disease be treated?

Because Danon disease can be associated with rapidly progressive cardiomyopathy and sudden death, careful monitoring of heart disease is required. Aggressive interventions may be recommended for people showing signs of progressive heart failure (e.g., early intervention with heart transplantation or implantable cardioverter-defibrillator).[1][2] However, the severity of cardiomyopathy does vary, particularly in females. Management will depend on the presence and severity of the heart disease, and will be tailored to the needs of the patient.
Last updated: 3/19/2010

References
  1. Yang Z et al. Danon disease as an underrecognized cause of hypertrophic cardiomyopathy in children. Circulation. 2005;
  2. Maron BJ et al. Clinical outcome and phenotypic expression in LAMP2 cardiomyopathy. JAMA. 2009;


GARD Video Tutorial

  • Finding Treatment Information - A video developed by GARD Information Specialists that explains how you can find information about treatment for a rare disease.

    Finding Treatment Information

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.