Article
The Way Forward for Mechanism-Based Therapeutics in Genetically Defined Neurodevelopmental Disorders.
Clinical pharmacology and therapeutics - 1 Oct 2018
Modi Meera E, Sahin Mustafa
Abstract excerpt
Rare genetically defined neurodevelopmental disorders with increased risk of autism have recently become an entry point for autism-related drug discovery. Through exploration of downstream effects of the pathological mutations, specific mechanistic pathways have been identified as dysregulated. The identification of shared mechanisms across forms of autism opens the door for the development of novel...
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