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Neurodevelopmental Disorders and Treatment

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Neurodevelopmental Framework for Drug Discovery Neurodevelopmental Disorders

Author(s): Stephen White, Russell Clark

There has been a revolution in genetic findings in neurodevelopmental disorders (NDDs) during the last decade, with several discoveries crucial to understanding their aetiology and pathophysiology. Clinical studies with single-gene disorders like fragile X syndrome show how difficult it is to find new treatment targets in NDDs. Incorporating a developmental viewpoint into the drug development process for NDDs should aid in overcoming some of the present challenges in identifying and evaluating new treatments. This publication summarises the proceedings of the European College of Neuropsychopharmacology's 'New Frontiers Meeting' on neurodevelopmental diseases, which was held in collaboration with the AIMS-2-TRIALS project, which is funded by the Innovative Medicines Initiative. It brought together academic and industrial professionals in developmental genetics, autism, neurodevelopmental disorders, and therapeutic trials, as well as regulators, patient and family organisations, and other stakeholders. From a neurodevelopmental standpoint, the meeting aimed to provide a platform for concentrated exchange on scientific insights, difficulties, and techniques that might be applied to the development of CNS treatments. Multidisciplinary translational consortia that produce basic and clinical research at the same time could be crucial in furthering knowledge in the sector. Although it is commonly agreed that clinical trials for NDDs in children are necessary, safety concerns should influence every aspect of their design. With regulatory backing, industry and academics should collaborate to enhance knowledge of the biology of brain development, determine the ideal timing of interventions, and translate these results into novel pharmaceuticals that meet the requirements of users and families.

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