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Significance Familial dysautonomia (FD) is a fatal genetic disorder that disrupts development of the peripheral nervous system (PNS) and causes progressive degeneration of the PNS and retina, ultimately leading to blindness. The underlying cellular mechanisms responsible for neuronal death in FD have been elusive. Using a mouse model that recapitulates impaired PNS development, we report here that developing peripheral neurons die in FD as the result of disruptions in mitochondrial and actin function. Cell death can be prevented in vivo by the small molecule BGP-15. Given that disrupted mitochondrial function appears to be a hallmark of neurodegenerative diseases, future studies on the efficacy of BGP-15 for mitigating neuronal loss in other disorders is warranted.

BGP-15 prevents the death of neurons in a mouse model of familial dysautonomia