Manifesting heterozygotes in a Japanese family with a novel mutation in the muscle-specific phosphoglycerate mutase (PGAM-M) gene

Neuromuscul Disord. 1999 Oct;9(6-7):399-402. doi: 10.1016/s0960-8966(99)00039-5.

Abstract

Muscle-specific phosphoglycerate mutase (PGAM-M) deficiency results in a metabolic myopathy (glycogenosis type X). Three mutations in the PGAM-M gene have been described thus far, two in African-American families and one in a Caucasian family. In two of them, manifesting heterozygotes were documented. We found a new PGAM-M mutation in a Japanese family with partial PGAM deficiency: a G-to-A transition at nucleotide position 209, resulting in the substitution of a highly conserved glycine at codon 97 with aspartic acid (G97D). Two heterozygous family members for the G97D mutation presented with exercise intolerance and muscle cramps. We describe the first PGAM-M mutation in the Japanese population and confirm that heterozygous individuals can be symptomatic.

Publication types

  • Case Reports

MeSH terms

  • Amino Acid Sequence
  • Amino Acid Substitution
  • Asian People / genetics
  • Black People
  • Female
  • Genetic Carrier Screening*
  • Glycogen Storage Disease / enzymology
  • Glycogen Storage Disease / genetics*
  • Humans
  • Japan
  • Male
  • Middle Aged
  • Molecular Sequence Data
  • Muscle, Skeletal / enzymology*
  • Mutation, Missense*
  • Pedigree
  • Phosphoglycerate Mutase / chemistry
  • Phosphoglycerate Mutase / genetics*
  • Polymerase Chain Reaction
  • Sequence Alignment
  • White People

Substances

  • Phosphoglycerate Mutase