title: Potential of fibrates in the treatment of fatty acid oxidation disorders: revival of classical drugs?
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.
authors.affiliation: INSERM U393, Hôpital Necker, Tour Lavoisier, 149 rue de Sèvres, 75015, Paris, France.
authors.forename: F
authors.initials: F
authors.lastname: Djouadi
authors.forename: F
authors.initials: F
authors.lastname: Aubey
authors.forename: D
authors.initials: D
authors.lastname: Schlemmer
authors.forename: S
authors.initials: S
authors.lastname: Gobin
authors.forename: P
authors.initials: P
authors.lastname: Laforet
authors.forename: R J A
authors.initials: RJ
authors.lastname: Wanders
authors.forename: A W
authors.initials: AW
authors.lastname: Strauss
authors.forename: J P
authors.initials: JP
authors.lastname: Bonnefont
authors.forename: J
authors.initials: J
authors.lastname: Bastin
mesh_terms: D044942:Acyl-CoA Dehydrogenase, Long-Chain / Q000096:biosynthesis / Q000172:deficiency / Q000235:genetics; D001629:Bezafibrate / Q000494:pharmacology; D002334:Carnitine O-Palmitoyltransferase / Q000096:biosynthesis / Q000172:deficiency / Q000235:genetics; D002478:Cells, Cultured; D002995:Clofibric Acid / Q000494:pharmacology* / Q000627:therapeutic use; D004790:Enzyme Induction / Q000187:drug effects; D005227:Fatty Acids / Q000378:metabolism; D005347:Fibroblasts / Q000187:drug effects* / Q000201:enzymology; D006801:Humans; D000960:Hypolipidemic Agents / Q000494:pharmacology* / Q000627:therapeutic use; D008052:Lipid Metabolism, Inborn Errors / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D008928:Mitochondria / Q000187:drug effects / Q000201:enzymology; D010084:Oxidation-Reduction / Q000187:drug effects; D047495:PPAR gamma / Q000819:agonists* / Q000378:metabolism; D012333:RNA, Messenger / Q000096:biosynthesis
chemical_list: D005227:Fatty Acids; D000960:Hypolipidemic Agents; D047495:PPAR gamma; D012333:RNA, Messenger; D002995:Clofibric Acid; D044942:Acyl-CoA Dehydrogenase, Long-Chain; D002334:Carnitine O-Palmitoyltransferase; D001629:Bezafibrate