Screening of late-onset Pompe disease in a sample of Mexican patients with myopathies of unknown etiology: identification of a novel mutation in the acid alpha-glucosidase gene

J Child Neurol. 2010 Aug;25(8):1034-7. doi: 10.1177/0883073809356035. Epub 2010 Mar 29.

Abstract

Pompe disease or glycogen-storage disease type 2 (GSD2, OMIM 232300) is an autosomal recessive disorder caused by mutations in the acid alpha-glucosidase gene. Late-onset GSD2 resembles some limb-girdle and Becker muscular dystrophies. The screening of GSD2 through the measurement of acid alpha-glucosidase activity in dried blood spots was applied to a selected sample of 5 Mexican patients with proximal myopathies of unknown etiology. Only 1 male patient showed a low level of acid alpha-glucosidase activity and a compound heterozygote genotype for the c.-32-13T>G splicing mutation present in most white late-onset Pompe disease cases and the novel mutation p.C558S. To our knowledge, this is the first report of a Mexican patient with late-onset GSD2. The identification of c.-32-13T>G in our patient could reflect the genetic contribution of European ancestry to the Mexican population. The enzymatic screening of GSD2 could be justified in patients with myopathies of unknown etiology.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Age of Onset
  • Female
  • Genetic Predisposition to Disease / genetics
  • Genetic Testing / methods
  • Genotype
  • Glycogen Storage Disease Type II / enzymology*
  • Glycogen Storage Disease Type II / genetics*
  • Glycogen Storage Disease Type II / physiopathology
  • Humans
  • Male
  • Mexico / ethnology
  • Muscular Diseases / enzymology*
  • Muscular Diseases / genetics*
  • Muscular Diseases / physiopathology
  • Mutation / genetics*
  • White People / ethnology
  • White People / genetics
  • alpha-Glucosidases / genetics*

Substances

  • GAA protein, human
  • alpha-Glucosidases