The main findings of the present study were as follows: (1) the level of miR-184 is associated with AFD and is also modulated by ERT; (2) a higher circulating level of miR-184 is independently associated with a lower risk of cardiac damage in AFD patients; and (3) adding miR-184 to a comprehensive clinical model improves the prediction of cardiac damage in AFD patients. Here, ELF3 is linked to Nager acrofacial dysostosis.