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- W3206819141 abstract "Breakthroughs in treatments for neurogenic forms of intellectual disability disorder (IDD) have not widely materialized in the clinic despite extensive research in preclinical mechanisms in rodent models. We will discuss various aspects in syndromic IDD: 1) past barriers and failures in treatment discovery; 2) corrective approaches in translational biomarker development; and 3) promising novel individualized treatment approaches including nonmedical, gene therapy, brain stimulation, and pharmacotherapy. Data on translational and individualized biomarker and treatment approaches will be obtained from systematic PubMed searches of published studies within the last decade. In addition, the clinician will present relevant clinical cases and draw from his extensive experience involved in 2 large NIH translational medicine programs for fragile X syndrome (FXS) and tuberous sclerosis (TS). 1) Successful efforts in animal models of neurogenic syndromes have not translated easily to clinical success. 2) Innovative approaches to more closely tie mouse and human work in early stages have begun to uncover promising conserved biomarkers. 3) In addition to evidence-based standard-of-care treatments, several emerging areas of individualized disease-modifying treatment approaches in TS and FXS have begun to set the stage for a new era of individualized transformational treatments in syndromic IDD. In clinical work, it is common knowledge that individuals with IDD, even in neurogenic syndromes, are highly heterogenous in presentation and that drug response is highly variable. In this session, we carefully consider how individualized treatment approaches can begin to account for stochastic and experience-driven processes of brain development in humans by more closely ensuring a degree of objective parallelism with the rodent models they represent. Several examples of successful breakthroughs in this area are discussed." @default.
- W3206819141 created "2021-10-25" @default.
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- W3206819141 date "2021-10-01" @default.
- W3206819141 modified "2023-09-26" @default.
- W3206819141 title "8.4 HURTLING TOWARD PRECISION MEDICINE IN SYNDROMIC INTELLECTUAL DISABILITY DISORDERS: INSIGHTS FROM MOUSE TO MAN" @default.
- W3206819141 doi "https://doi.org/10.1016/j.jaac.2021.07.547" @default.
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