Lissencephaly is a spectrum of rare, genetic disorders in which the brain fails to develop its hallmark folds. The disorders are often associated with seizures and intellectual disability and currently there are no available treatments. A new study, however, has identified a molecular mechanism that underlies some lissencephaly disorders -- and a drug that prevents and reverses lissencephaly malformations in organoids (small, three-dimensional replicas of developing brains that allow scientists to study early brain development).
