Childhood onset limb-girdle muscular dystrophies in the Aegean part of Turkey

Acta Myol. 2018 Sep 1;37(3):210-220. eCollection 2018 Sep.

Abstract

The aim of this study is to analyze the epidemiology of the clinical and genetic features of childhood-onset limb-girdle muscular dystrophies (LGMD) in the Aegean part of Turkey. In total fifty-six pediatric cases with LGMD followed in four different pediatric neurology departments in the Aegean region of Turkey were evaluated. Among them, LGMD2C was the most common followed by LGMD2A, LGMD2D, and LGMD2F with equal frequencies. In twenty-eight patients (50%) the diagnosis could be confirmed by genetic analysis, where SGCG proved to be disease-causing in most of the cases. About half of the patients were diagnosed with whole exome or targeted gene sequencing. A positive correlation between muscle biopsy and genetic findings were observed in 11% of the patients. We report one novel frameshifting mutation in TTN. Knowledge on frequencies of childhood-onset limb-girdle muscular dystrophies and related genes in Turkey will lead to a prompt diagnosis of these neuromuscular disorders.

Keywords: Turkey; childhood; genetic diagnosis; limb-girdle muscular dystrophy.

MeSH terms

  • Adolescent
  • Age of Onset
  • Biopsy
  • Calpain / genetics
  • Child
  • Child, Preschool
  • Connectin / genetics
  • Female
  • Genetic Testing
  • Humans
  • Infant
  • Lamin Type A / genetics
  • Male
  • Mannosyltransferases / genetics
  • Microfilament Proteins
  • Muscle Proteins / genetics
  • Muscle, Skeletal / pathology
  • Muscular Dystrophies, Limb-Girdle / complications
  • Muscular Dystrophies, Limb-Girdle / epidemiology*
  • Muscular Dystrophies, Limb-Girdle / genetics*
  • Muscular Dystrophies, Limb-Girdle / pathology
  • Sarcoglycanopathies / epidemiology
  • Sarcoglycanopathies / genetics
  • Sarcoglycans / genetics
  • Turkey / epidemiology

Substances

  • Connectin
  • LMNA protein, human
  • Lamin Type A
  • MYOT protein, human
  • Microfilament Proteins
  • Muscle Proteins
  • SGCB protein, human
  • Sarcoglycans
  • TTN protein, human
  • Mannosyltransferases
  • protein O-mannosyltransferase
  • CAPN3 protein, human
  • Calpain

Supplementary concepts

  • Limb-girdle muscular dystrophy type 2A
  • Limb-girdle muscular dystrophy type 2F
  • Limb-girdle muscular dystrophy, type 2C