Limb-girdle muscular dystrophy type 2A in Brazilian children

Arq Neuropsiquiatr. 2015 Dec;73(12):993-7. doi: 10.1590/0004-282X20150168.

Abstract

Calpainopathy is an autosomal recessive limb girdle muscular dystrophy (LGMD2A) caused by mutations in CAPN3 gene.

Objective: To present clinical and histological findings in six children with a molecular diagnosis of LGMD2A and additionally the MRI findings in two of them.

Method: We retrospectively assessed medical records of 6 patients with mutation on CAPN3 gene.

Results: All patients were female (three to 12 years). The mean of age of disease onset was 9 years. All of them showed progressive weakness with predominance in lower limbs. Other findings were scapular winging, joint contractures and calf hypertrophy. One female had a more severe phenotype than her dizygotic twin sister that was confirmed by muscle MRI. Muscle biopsies showed a dystrophic pattern in all patients.

Conclusion: In this cohort of children with LGMD2A, the clinical aspects were similar to adults with the same disorder.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Brazil
  • Child
  • Child, Preschool
  • DNA Mutational Analysis
  • Female
  • Humans
  • Muscular Dystrophies, Limb-Girdle / genetics*
  • Muscular Dystrophies, Limb-Girdle / pathology*
  • Mutation / genetics
  • Phenotype
  • Retrospective Studies

Supplementary concepts

  • Limb-girdle muscular dystrophy type 2A