To the Editor: Carnitine palmitoyltransferase II (CPT2) deficiency is a rare autosomal recessive disorder of mitochondrial fatty acid oxidation. The most common form of this disorder is characterized by muscle stiffness, myalgia, and exercise intolerance,¹,² and current dietary approaches often do not prevent attacks of rhabdomyolysis. We found that bezafibrate, a commonly used hypolipidemic drug,³ restored the capacity for normal fatty acid oxidation in muscle cells from patients with a mild form of CPT2 deficiency by stimulating the expression of the mutated gene.⁴

We evaluated the efficacy of bezafibrate as a treatment for the mild form of CPT2 deficiency . . .