Daily oral treatment of CMT1A rats with PXT3003 down-regulates Pmp22 expression and reduces signs of motor and sensory neuropathy. (A) A 9-week treatment with PXT3003 (BCL 30 μg/kg, NTX 3.5 μg/kg and SRB 1.05 mg/kg) decreased Pmp22 to Mpz mRNA ratio in the sciatic nerve. n = 18, 20 and 18 animals for respectively WT vehicle, TG vehicle and TG PXT3003 groups. (B) Bar test. The latency to fall for transgenic rats was significantly improved after 9 weeks of treatment with PXT3003. (C) Kaplan-Meier representation of the data set in (B) demonstrated a positive effect of PXT3003. (D) After 9 weeks of treatment, the inclined plane score was significantly improved for transgenic rats. (B) to (D) n = 36, 38 and 36 animals for respectively WT vehicle, TG vehicle and TG PXT3003 groups. ( E ) A 4-month long PXT3003 treatment normalised the impaired thermal sensitivity of transgenic rats in the hot plate test. n = 12, 10 and 7 animals respectively for WT, vehicle, TG vehicle and TG PXT3003 groups. *P < 0.05; **P < 0.01; ***P < 0.001 vs TG vehicle; ANOVA with Dunnett’s test (except in (C), logrank). Data are shown as mean + SEM.