Neurology

Patient sample and mouse studies suggest positive allosteric modulators (PAMs) of CHRM4 could help treat Rett syndrome. In postmortem cerebellum tissue samples from Rett syndrome patients, levels of CHRM4 were lower than in samples from unaffected subjects. In a mouse model of Rett syndrome, a tool compound CHRM4 PAM increased scores in associative learning and memory tasks compared with vehicle. Next steps could include testing PAMs of CHRM4 in additional animal models of Rett syndrome...