Clinical manifestations and MRI features of Danon disease: a case series

BMC Cardiovasc Disord. 2023 Aug 11;23(1):397. doi: 10.1186/s12872-023-03356-y.

Abstract

Background: Danon disease (DD) is an exceptionally uncommon X-linked dominant lysosomal glycogen storage disorder characterized by pronounced ventricular hypertrophy and cardiac insufficiency. The timely identification of cardiac impairment in individuals with DD holds significant clinical importance.

Case presentation: We present a case of Danon Disease in a three-generation pedigree from Anhui Province, China. Clinical features and laboratory data were collected and analyzed for a 16-year-old male proband (III-1) and two affected female family members (II-2 and II-3). The proband exhibited Wolf-Parkinson-White syndrome, hypertrophic cardiomyopathy, abnormal cognitive function, and muscle weakness. Gene sequencing confirmed a mutation (c.963G > A) in the LAMP-2 gene.

Conclusion: Patients with DD may present both dilated and hypertrophic cardiomyopathy. Comprehensive myocardial tissue characterization by MRI plays a key role in the diagnosis of the disease.

Keywords: Danon disease; Extracellular volume fraction; Magnetic resonance imaging; T1 mapping.

Publication types

  • Case Reports

MeSH terms

  • Cardiomyopathy, Hypertrophic* / diagnostic imaging
  • Cardiomyopathy, Hypertrophic* / genetics
  • Female
  • Glycogen Storage Disease Type IIb* / diagnosis
  • Glycogen Storage Disease Type IIb* / genetics
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Mutation
  • Wolff-Parkinson-White Syndrome* / diagnostic imaging
  • Wolff-Parkinson-White Syndrome* / genetics