Journal of Inherited Metabolic Disease

, Volume 29, Issue 2, pp 341–342

Potential of fibrates in the treatment of fatty acid oxidation disorders: Revival of classical drugs?

Authors

  • F. Djouadi
    • INSERM U393Hôpital Necker
  • F. Aubey
    • INSERM U393Hôpital Necker
  • D. Schlemmer
    • INSERM U393Hôpital Necker
  • S. Gobin
    • INSERM U393Hôpital Necker
  • P. Laforet
    • Institut de MyologieHôpital La Pitié-Salpétrière
  • R. J. A. Wanders
    • Laboratory for Genetic Metabolic DiseasesAcademic Medical Centre
  • A. W. Strauss
    • Vanderbilt Children's Hospital
  • J. P. Bonnefont
    • INSERM U393Hôpital Necker
    • INSERM U393Hôpital Necker
SSIEM SYMPOSIUM 2005

DOI: 10.1007/s10545-006-0266-3

Cite this article as:
Djouadi, F., Aubey, F., Schlemmer, D. et al. J Inherit Metab Dis (2006) 29: 341. doi:10.1007/s10545-006-0266-3

Summary

Exposure to fibrates leads to normalization of fatty acid oxidation (FAO) in fibroblasts from patients with myopathic forms of CPT2 deficiency or VLCAD deficiency. Correction of FAO is related to a drug-induced increase of residual enzyme activity, and this could provide a new treatment strategy for these disorders.

Copyright information

© SSIEM and Springer 2006