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