[Distal myopathy due to mutations of GNE gene: clinical spectrum and diagnosis]

Rev Neurol (Paris). 2008 May;164(5):434-43. doi: 10.1016/j.neurol.2008.02.040. Epub 2008 Apr 15.
[Article in French]

Abstract

Distal myopathies are rare muscular disorders clinically characterized by a predominantly distal muscular involvement. Among recessive forms, the myopathy resulting from mutations in the UDP-N-acetylglucosamine-2-epimerase/N-acetylmannosamine kinase (GNE) gene, often designated as Nonaka myopathy, primarily affect young adults and are characterized by muscle wasting and weakness predominating on the anterior compartment of the leg, a remarkable quadriceps sparing and a frequent evolution towards ambulation loss after a few years. Finding rimmed vacuoles on muscle biopsy is a further argument for the diagnosis. However, the presentation and course may vary and we describe four patients who illustrate the clinical spectrum of the disease: the first patient had a classical form with progressive weakness over several years, the second one a rapidly progressive myopathy leading to ambulation loss within three years from onset, the third one a very slow course with no ambulation loss after several decades, and the last one a progressive form with misleading neurogenic features on the EMG. One of our four patients harbored a homozygous mutation, and three others were compound heterozygous, two of them displaying an original mutation: one had a c.2036 T>G (p.Val679Gly) substitution, the c.829 C>T (p.Arg277Cys) substitution.

Publication types

  • Case Reports
  • English Abstract

MeSH terms

  • Adipose Tissue / pathology
  • Adult
  • Aged
  • Distal Myopathies / diagnosis
  • Distal Myopathies / genetics*
  • Distal Myopathies / pathology
  • Electromyography
  • Female
  • Humans
  • Leg / pathology
  • Male
  • Middle Aged
  • Muscle Fibers, Skeletal / pathology
  • Muscle, Skeletal / pathology
  • Mutation
  • Phosphotransferases (Alcohol Group Acceptor) / genetics*
  • Tendons / pathology
  • Tomography, X-Ray Computed
  • Wasting Syndrome / pathology

Substances

  • Phosphotransferases (Alcohol Group Acceptor)
  • N-acylmannosamine kinase