A Blueprint for Translational Precision Medicine in Autism Spectrum Disorder and Related Neurogenetic Syndromes
Thom R, Warren T, Khan S, Muhle R, Wang P, Brennand K, Zürcher N, Veenstra-VanderWeele J, Hoffman E. A Blueprint for Translational Precision Medicine in Autism Spectrum Disorder and Related Neurogenetic Syndromes. Journal Of Child And Adolescent Psychopharmacology 2025, 35: 178-193. PMID: 40138183, PMCID: PMC12344128, DOI: 10.1089/cap.2025.0023.Peer-Reviewed Original ResearchConceptsAutism spectrum disorderNeurogenetic syndromesSpectrum disorderNeurobiology of autism spectrum disordersRett syndromeTarget engagementEvaluation of target engagementPlacebo responseEvaluate target engagementClinical trialsClinical trial design strategiesIntermediate phenotypesPreclinical model systemsTreatment discoveryObjectives:</i></b>Trial design strategiesTrofinetideMolecular targetsNeurobiologyFace validityHuman clinical trialsPrecision medicineStem cell model systemsPrecision medicine approachIdentification of molecular targets
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