Neurology

In vitro and mouse studies suggest the Hsp72 inducer BGP-15 could help treat familial dysautonomia, a fatal disorder often caused by a point mutation in the IKBKAP gene. In primary IKBKAP-knockout mouse neurons, the compound decreased levels of reactive oxygen species (ROS) and cell death compared with vehicle. In a genetic mouse model of familial dysautonomia, the compound decreased neuropathy and cardiac innervation defects, a marker of the disease. Next steps could include clinical testing of BGP-15 in patients with familial dysautonomia...