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?


  • 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

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


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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© SSIEM and Springer 2006