Why the study?
Clinical manifestations of Danon disease vary between sexes, with men presenting earlier with severe hypertrophic cardiomyopathy and arrhythmias, while women present with later progression and less homogeneous symptoms.
Danon disease is a rare genetic disorder with heterogeneous clinical presentations, and genetic testing is crucial for diagnosis and family screening, with heart transplantation being an effective treatment for advanced heart failure.
Sex differences in Danon cardiomyopathy severity may guide monitoring; leaves open prospective trials for targeted therapies.
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Romero et al. (2025) studied this question.
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