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

F. Djouadi 1, 2 F. Aubey 2 D. Schlemmer 2 S. Gobin 2 P. Laforet 3 R.J.A. Wanders 4 A.W. Strauss 5 J. Bonnefont 2 J. Bastin 2 
1 CRC (UMR_S_1138 / U1138) - Centre de Recherche des Cordeliers
2 Inserm U393 - Handicaps génétiques de l'enfant
3 CHU Pitié-Salpêtrière [AP-HP]
4 AMC - Academic Medical Center - Academisch Medisch Centrum [Amsterdam]
5 Vanderbilt University [Nashville]
Abstract : 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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https://www.hal.inserm.fr/inserm-02896275
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F. Djouadi, F. Aubey, D. Schlemmer, S. Gobin, P. Laforet, et al.. Potential of fibrates in the treatment of fatty acid oxidation disorders: Revival of classical drugs?. Journal of Inherited Metabolic Disease, Springer Verlag, 2006, 29 (2-3), pp.341-342. ⟨10.1007/s10545-006-0266-3⟩. ⟨inserm-02896275⟩

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