DanonDisease.org

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Welcome to DanonDisease.org

Danon Disease is a rare genetic condition causing muscle weakness (muscular dystrophy), heart disease (cardiomyopathy), and mental retardation (or learning problems).

Danon Disease was first described in 1981, and affects males more severely than females. Boys will often develop symptoms in childhood or adolescence, while in females the symptoms may not be apparent until adolescence or adulthood. Although males have an earlier and more rapidly progressive disease, serious medical problems can occur in females.

The disease is NOT well-understood and physicians are largely unfamiliar with Danon Disease. In fact, most physicians probably had not even heard of Danon Disease. We (the medical commmunity) do not yet know how many people and families are affected with Danon Disease.

  • ABOUT THIS SITE: This website was started by a physician-researcher (Matthew Taylor MD, PhD) in 2006. Dr. Taylor has developed a clinical and research interest in this rare genetic condition.
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  • Our goals are to help Danon Disease patients and families by:

  • Providing basic information about Danon Disease
  • Explaining how Danon Disease is inherited and how it can be passed-on to other family members.
  • Identifying other families with Danon Disease, with the goal of starting a 'Disease Registry'.
  • Finding patients and families who wish to participate in research to understand (and ultimately to treat) Danon Disease.

If you or someone in your family has Danon Disease, please consider contacting us to share your story so we can learn more about how Danon Disease is affecting patients and families.  If you would like to participate in our Danon Disease research project, please email Dr. Taylor.
 
2019 Danon Disease Research Activities
-A Gene Therapy Trial for Danon Patients (starting in 2019) (
LINK
)