Pathways of Neurodevelopmental Disorders (C8)

Advances in genetics and animal models for many neurodevelopmental disorders have led to the development of targeted treatments that can reverse the neurobiological abnormalities in the animal models. Human studies have begun to show significant benefit from targeted treatments for several neurodevelopmental disorders including fragile X syndrome, tuberous sclerosis, Angelman Syndrome, Down Syndrome, Rett Syndrome and neurofibromatosis with the potential for many more. There are remarkable commonalities in the dysfunction of key pathways and the molecular mechanisms involved in synaptic plasticity across neurodevelopmental disorders. This Keystone Symposia meeting will cover the advances of targeted treatments in several neurodevelopmental disorders with an emphasis on common pathways across disorders and treatments that may help more than one disorder. This conference will also address the gap of how environmental factors including seizures and toxins can impact genetically vulnerable populations such as those with neurodevelopmental problems and the molecular mechanisms including mitochondrial dysfunction, oxidative stress, miRNA dysregulation and neuroinflammation that are involved. The goal of this conference is to stimulate new treatments and biomarkers to assess treatments in neurodevelopmental disorders and to stimulate early intervention with prophylactic and targeted treatments. This conference is innovative because it pulls together basic science and translational clinical trials. By going from bench to bedside and mixing clinicians with molecular and neurobiologists we will stimulate new ideas for treatment that will be beneficial across the spectrum of neurodevelopmental disorders.