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Eur J Med Genet
February 2020
Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata and IRCAB Foundation, Udine, Italy.
Danon disease is characterized by hypertrophic cardiomyopathy, skeletal myopathy, and intellectual disability due to deficiency of the lysosome-associated membrane protein-2 (LAMP-2). Although heart transplantation is considered an option for end stage Danon cardiomyopathy, scarce information is available about long term follow up. We report on long term follow up (14.
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