PXL770 treatment improves VLCFA levels in spinal cord, axonal morphology of sciatic nerve and locomotor function in Abcd1 KO mice. 13-month-old Abcd1 KO mice were treated with PXL770 75 mg/kg (BID oral) for 12 weeks. (A) VLCFA content measured by LC-MS in spinal cord. Data are mean ± SEM, n = 8 animals/condition. (B) Axonal morphology of myelin and neurons determined by morphometric analysis of transversal slices of the sciatic nerve by EM (magnification 800X). Data are mean ± SEM, n = 4 animals per condition. Behavioral and locomotor function assessment by (C) open field test and (D) Balance beam test. Data are median ± 95% interval confidence, n = 8 animals for wild-type and KO untreated, 7 animals for PXL770. *P < 0.05, **P < 0.01, ***P < 0.001 ****P < 0.0001 (Dunnett’s test versus untreated).