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The Way Forward for Mechanism‐Based Therapeutics in Genetically Defined Neurodevelopmental Disorders
Author(s) -
Modi Meera E.,
Sahin Mustafa
Publication year - 2018
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.1181
Subject(s) - mechanism (biology) , neuroscience , computational biology , medicine , biology , epistemology , philosophy
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 “mechanism‐based therapeutics.” However, confidence in the therapeutic mechanism does not diminish the need for well‐designed clinical trials.