Phenotypic Duchenne muscular dystrophy with C-terminal domain

Pediatr Neurol. 1992 Jul-Aug;8(4):310-2. doi: 10.1016/0887-8994(92)90373-7.

Abstract

We report a patient with X-linked muscular dystrophy who had rapidly progressive muscle weakness and became wheelchair-bound at age 10 years. Clinically, he was diagnosed as having Duchenne muscular dystrophy; however, he was diagnosed as having Becker muscular dystrophy by dystrophin tests using a C-terminal monoclonal antibody. No immunolabelling was observed with a monoclonal antibody against the N-terminal domain. Multiplex polymerase chain reaction analysis revealed the deletion of exons 3-19. The data suggest that the deletion of the N-terminal domain of dystrophin can cause a severe phenotype even when the C-terminus of the protein is well preserved.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Antibodies, Monoclonal
  • Biopsy
  • Chromosome Deletion*
  • Dystrophin / genetics*
  • Exons / genetics
  • Genetic Linkage / genetics*
  • Humans
  • Immunoblotting
  • Male
  • Muscles / pathology
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / pathology
  • Phenotype*
  • Polymerase Chain Reaction
  • Sex Chromosome Aberrations / genetics*
  • Sex Chromosome Aberrations / pathology
  • Terminator Regions, Genetic / genetics*
  • X Chromosome*

Substances

  • Antibodies, Monoclonal
  • Dystrophin