Unusual clinical expression of dystrophinopathy in a female, mimicking a congenital myopathy

Eur Neurol. 1999;42(4):221-4. doi: 10.1159/000008111.

Abstract

A 25-year-old woman with negative family history and delayed motor development presented hypotrophy of the right lower limb and calf hypertrophy since age 7 and she complained of muscle weakness since 23. Neurological examination showed a thin elongated face, high-arched palate, high-pitched voice, proximal wasting and weakness, impairment of distal muscles in the lower limbs. CK was 3, 034 U/l, EMG showed a myopathic pattern. Muscle biopsy displayed dystrophic features with diffuse dystrophin deficiency; immunoblotting demonstrated quantitative reduction of the protein and normal molecular weight. Lyonization study showed skewed X-inactivation with the maternal X active. Seven years' follow-up did not show progression of the disease.

Publication types

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

MeSH terms

  • Adult
  • Biopsy
  • Disease Progression
  • Dystrophin / deficiency*
  • Dystrophin / genetics*
  • Female
  • Gene Expression Regulation
  • Genetic Carrier Screening / methods
  • Heterozygote
  • Humans
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / pathology
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / metabolism*
  • Muscular Dystrophies / pathology
  • Sex Factors

Substances

  • Dystrophin

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