Danon disease: Gender differences in presentation and outcomes

Background Danon disease (DD) is a rare X-linked autophagic vacuolar myopathy, characterized by high penetrance and severe cardiomyopathy. Because of its rarity, the natural history (NH) is uncertain. Objectives We aimed to describe disease variability and outcomes through a systematic review of all published DD cases. Methods Among 83 manuscripts in MEDLINE and EMBASE on DD cases published until October 2017, we identified 146 patients with...