Muskelveränderungen PMP22-transgener („CMT“-) Ratten unter Therapie mit dem rekombinanten Medikament PXT3003

The treatment of CMT1A, the most common hereditary sensorimotor polyneuropathy, has so far been limited to symptomatic approaches, which is why many affected individuals develop significant physical impairments over the course of the disease. The combined drug PXT3003 consisting of naltrexone, D-sorbitol and baclofen offers a causal therapeutic approach while maintaining a low side-effect profile due to the synergistic effect of the three different substances, each administered at subtherapeutic doses. The aim of this study was to investigate the effects of CMT1A and the impact of treatment with PXT3003 on muscle tissue in an established rat model of CMT1A. Evidence of neurogenic muscle damage was observed, including alterations in muscle fiber type composition in the muscles of rats affected by CMT1A. Treatment with PXT3003 reduced the extent of neurogenic damage and resulted in a more physiological muscle fiber type composition. In summary, positive changes in muscle histology following treatment with PXT3003 were demonstrated, which were also reflected in the phenotypic analyses. The safety of PXT3003 has already been confirmed in an international phase II and III clinical trial in patients with genetically confirmed CMT1A. A second phase III trial is ongoing to further confirm efficacy.