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Translating Precision Medicine for Autism Spectrum Disorder: A Pressing Need


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High genetic and clinical heterogeneity has rendered existing drug discovery efforts inefficient for patients with neurodevelopmental disorders, highlighting the need for omics- and endophenotyping-based approaches in patient diagnosis and treatment.

Autism spectrum disorder (ASD) is a heterogenous group of neurodevelopmental disorders (NDDs) with a high unmet medical need. Currently, ASD is diagnosed according to behavior-based criteria that overlook clinical and genomic heterogeneity, thus repeatedly resulting in failed clinical trials. Here, we summarize the scientific evidence pointing to the pressing need to create a precision medicine framework for ASD and other NDDs. We discuss the role of omics and systems biology to characterize more homogeneous disease subtypes with different underlying pathophysiological mechanisms and to determine corresponding tailored treatments. Finally, we provide recent initiatives towards tackling the complexity in NDDs for precision medicine and cost-effective drug discovery.

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