The clinical spectrum of sarcoglycanopathies

Neurology. 1999 Jan 1;52(1):176-9. doi: 10.1212/wnl.52.1.176.

Abstract

A group of 204 muscular dystrophy patients were screened for immunohistochemical and biochemical alpha-sarcoglycan defect and their DNA was analyzed for pathogenetic mutation in the four sarcoglycan genes. We identified 21 patients with alpha-, beta-, or gamma-sarcoglycan gene mutations. Patients with alpha-sarcoglycan gene mutations were clinically heterogeneous and showed either a rapid progressive or a late-onset slow course. In the slowly evolving group, a residual alpha-sarcoglycan protein was present, and its level correlated with a milder disease course and significant later inability to stand up from the floor (p < 0.00005). Most patients with beta- and gamma-sarcoglycan gene mutations presented a severe clinical course. There is a considerably different pattern of muscle involvement and disease course in these disorders, compared with dystrophinopathies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Cytoskeletal Proteins / genetics*
  • Disease Progression
  • Dystroglycans
  • Female
  • Genetic Testing
  • Humans
  • Male
  • Membrane Glycoproteins / genetics*
  • Middle Aged
  • Movement
  • Muscular Dystrophies / genetics*
  • Mutation
  • Neurologic Examination
  • Phenotype
  • Prognosis
  • Sarcoglycans
  • Severity of Illness Index

Substances

  • Cytoskeletal Proteins
  • DAG1 protein, human
  • Membrane Glycoproteins
  • Sarcoglycans
  • Dystroglycans

Grants and funding