Novel Rod Domain Duplication in Dystrophin Resulting in X-Linked Dilated Cardiomyopathy

Pediatr Neurol. 2015 Nov;53(5):439-41. doi: 10.1016/j.pediatrneurol.2015.07.008. Epub 2015 Jul 26.

Abstract

Background: X-linked dilated cardiomyopathy is a rare, cardio-specific form of dystrophinopathy allelic to Duchenne and Becker muscular dystrophy that results in heart failure without skeletal muscle weakness.

Patient description: We describe a previously healthy 16-year-old boy who presented with palpitations progressing to heart failure who was ultimately found to have a novel duplication of exons 13-16 in the dystrophin gene resulting in diagnosis of X-linked dilated cardiomyopathy.

Results: The patient was diagnosed with X-linked dilated cardiomyopathy through clinical diagnosis and genetic testing.

Conclusion: X-linked dilated cardiomyopathy shares genotypic overlap with Duchenne and Becker muscular dystrophy, with its distinctive feature being a lack of progressive muscular weakness.

Keywords: Becker muscular dystrophy; Duchenne muscular dystrophy; X-linked; cardiomyopathy; dystrophinopathy.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Cardiomyopathy, Dilated / diagnosis*
  • Cardiomyopathy, Dilated / diagnostic imaging
  • Cardiomyopathy, Dilated / genetics*
  • Cardiomyopathy, Dilated / physiopathology
  • Dystrophin / genetics*
  • Echocardiography
  • Electrocardiography
  • Exons
  • Heart / physiopathology
  • Humans
  • Male

Substances

  • DMD protein, human
  • Dystrophin

Supplementary concepts

  • Dmd-Associated Dilated Cardiomyopathy