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Updated: Feb 1, 2026

A Modified Method for Heterotopic Mouse Heart Transplantion
Published on: June 23, 2014
Heart transplantation in two adolescents with Danon disease
Daniel Oren1, Peter Chau2, Melanie Manning3
1Heart and Vascular Center, Semmelweis University, Budapest, Hungary.
Abstract:
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.
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