A white paper on a neurodevelopmental framework for drug discovery in autism and other neurodevelopmental disorders.

摘要: Abstract In the last decade there has been a revolution in terms of genetic findings neurodevelopmental disorders (NDDs), with many discoveries critical for understanding their aetiology and pathophysiology. Clinical trials single-gene such as fragile X syndrome highlight challenges investigating new drug targets NDDs. Incorporating developmental perspective into process development NDDs could help to overcome some current difficulties identifying testing treatments. This paper provides summary proceedings ‘New Frontiers Meeting’ on organised by European College Neuropsychopharmacology conjunction Innovative Medicines Initiative-sponsored AIMS-2-TRIALS consortium. It brought together experts genetics, autism, NDDs, clinical from academia industry, regulators, patient family associations, other stakeholders. The meeting sought provide platform focused communication scientific insights, challenges, methodologies that might be applicable CNS treatments perspective. Multidisciplinary translational consortia develop basic research parallel pivotal advance knowledge field. Although implementation paediatric populations is widely acknowledged essential, safety concerns should guide each aspect design. Industry join forces improve biology brain development, identify optimal timing interventions, translate these drugs, allowing needs users families, support regulatory agencies.