Journal of Inherited Metabolic Disease

, Volume 29, Issue 2–3, pp 341–342

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

  • F. Djouadi
  • F. Aubey
  • D. Schlemmer
  • S. Gobin
  • P. Laforet
  • R. J. A. Wanders
  • A. W. Strauss
  • J. P. Bonnefont
  • J. Bastin
SSIEM SYMPOSIUM 2005

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.

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Copyright information

© SSIEM and Springer 2006

Authors and Affiliations

  • F. Djouadi
    • 1
  • F. Aubey
    • 1
  • D. Schlemmer
    • 1
  • S. Gobin
    • 1
  • P. Laforet
    • 2
  • R. J. A. Wanders
    • 3
  • A. W. Strauss
    • 4
  • J. P. Bonnefont
    • 1
  • J. Bastin
    • 1
  1. 1.INSERM U393Hôpital NeckerParisFrance
  2. 2.Institut de MyologieHôpital La Pitié-SalpétrièreParisFrance
  3. 3.Laboratory for Genetic Metabolic DiseasesAcademic Medical CentreAmsterdamThe Netherlands
  4. 4.Vanderbilt Children's HospitalNashvilleUSA

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