Distal lipid storage myopathy due to PNPLA2 mutation

Neuromuscul Disord. 2008 Aug;18(8):671-4. doi: 10.1016/j.nmd.2008.06.382. Epub 2008 Jul 26.

Abstract

Distal myopathy is a group of heterogeneous disorders affecting predominantly distal muscles usually appearing from young to late adulthood with very rare cardiac complications. We report a 27-year-old man characterized clinically by distal myopathy and dilated cardiomyopathy, pathologically by lipid storage, and genetically by a PNPLA2 mutation. The patient developed weakness in his lower legs and fingers at age 20 years. Physical examination at age 27 years revealed muscle weakness and atrophy predominantly in lower legs and hands, and severe dilated cardiomyopathy. The patient had a homozygous four-base duplication (c.475_478dupCTCC) in exon 4 of PNPLA2.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Atrophy
  • Cardiomyopathy, Dilated / etiology
  • Cardiomyopathy, Dilated / genetics
  • Cardiomyopathy, Dilated / pathology
  • Distal Myopathies / genetics*
  • Distal Myopathies / pathology
  • Hand / pathology
  • Humans
  • Leg / pathology
  • Lipase / genetics*
  • Lipid Metabolism / genetics
  • Lipid Metabolism / physiology
  • Male
  • Mountaineering
  • Muscle Fibers, Skeletal / pathology
  • Muscle Weakness / etiology
  • Muscle, Skeletal / pathology
  • Mutation / genetics
  • Mutation / physiology

Substances

  • Lipase
  • PNPLA2 protein, human