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Figure 6 | Orphanet Journal of Rare Diseases

Figure 6

From: Polytherapy with a combination of three repurposed drugs (PXT3003) down-regulates Pmp22 over-expression and improves myelination, axonal and functional parameters in models of CMT1A neuropathy

Figure 6

Daily oral PXT3003 treatment of nerve-crushed mice restores nerve physiology, improves axonal and myelin integrity and functional behaviour. (A) A 21 and 30 day-treatment with PXT3003 (BCL 60 μg/kg, NTX 7 μg/kg and SRB 2.1 mg/kg) increased the amplitude of CMAP (measured in the gastrocnemius) of crushed nerves in male mice. n = 10 animals in each group. (B) to (D) A 42 day-treatment with PXT3003 significantly improved axonal calibre size (B), normalised the distribution of the number of myelinated axons in terms of axonal diameter (C) and improved the distribution of myelin g-ratio in tibial nerve cross sections (D). n = 6 for each group. (E) After 13 days of treatment, PXT3003 (BCL 600 μg/kg, NTX 70 μg/kg and SRB 21 mg/kg) improved significantly the functional activity of crushed mice as assessed by the paw surface pressure against the floor (paw surface bearing) of the affected paw normalised to the contralateral non-affected one. (F) to (H) In contrast to PXT3003, single drugs had no effect on surface bearing ratio. n = 10 for each group. *P < 0.05, **P < 0.01, ***P < 0.001 vs Crush Vehicle; t–test. Data are shown as mean ± SEM.

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