Danon disease (DD) is an X‐linked dominant disorder caused by a mutation in the lysosomal‐associated membrane protein‐2 (LAMP‐2) gene coding for the LAMP‐2 protein. We report two cases of successful heart transplantation (HT) in adolescent brothers with DD, including one who was bridged to HT for 34 days with a HeartWare left ventricular assist device. In both patients, the post‐transplant course was complicated by profound skeletal muscle weakness that resolved with corticosteroid withdrawal. These cases highlight that both HT and ventricular assist device support are feasible in patients with DD. Corticosteroid use may exacerbate skeletal myopathy, and therefore, steroid minimization may be warranted whenever possible.

The utility of hemodynamic ramp studies in single ventricle patients supported by continuous flow left ventricular assist devices (CF-LVADs) is largely unknown. We report the novel use of the hemodynamic ramp test to optimize a 26 year-old Fontan-palliated single-ventricle patient supported with a continuous-flow ventricular assist device (Heartware HVAD). Modest improvements in Fontan hemodynamics resulted in significant and persistent clinical improvement and symptomatic relief.

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