Developing new pharmacotherapies for autism
Open accessAbstract
Developing new pharmacotherapies for autism spectrum disorder (ASD) is a challenge. ASD has a complex genetic architecture, several neurobiological phenotypes and multiple symptom domains. However, new opportunities are emerging that could lead to the development of ‘targeted’ and individualized pharmacological interventions. Here, first we review these important new insights into the aetiology and neurobiology of ASD with particular focus on (i) genetic variants mediating synaptic structure and functioning and (ii) differences in brain anatomy, chemistry and connectivity in this condition. The characterization of the genotypic and phenotypic differences underlying ASD might in the future be invaluable for stratifying the large range of different individuals on the autism spectrum into genetically and/or biologically homogeneous subgroups that might respond to similar targeted interventions. Secondly, we propose a strategic framework for the development of targeted pharmacotherapies for ASD, which comprises several different stages in which research findings are translated into clinical applications. The establishment of animal models and cellular assays is important for developing and testing new pharmacological targets before initiating large‐scale clinical trials. Finally, we present the E uropean A utism I nterventions – A M ulticentre S tudy for D eveloping N ew M edications (EU‐AIMS) Initiative, which was set up in the context of the EU I nnovative M edicines I nitiative as the first E uropean platform for integrated translational research in ASD. The EU‐AIMS I nitiative consists of academic and industrial partners working in collaboration to deliver a more ‘personalized’ approach to diagnosing and treating ASD in the future.
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Brain Fires in Autism Spectrum Disorders
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Stratified medicine: methods for evaluation of predictive biomarkers
Malottki, K. (2017) · University of Birmingham Institutional Research Archive (University of Birmingham)
Making Visible The Lived Experiences of Mothers of Children with Autism Spectrum Disorder
Baird-Bate, K. (2019) · Queensland University of Technology · doi:10.5204/thesis.eprints.128644
Emerging Trends and Transdisciplinary Approaches in Autism Spectrum Disorder Management
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- Authors
- Ecker, C., Spooren, W., & Murphy, D.
- Affiliations
- Department of Forensic and Neurodevelopmental Sciences Institute of Psychiatry London UK
- F. Hoffmann-La Roche AG (CH)
- Corresponding author
- Ecker, C.
- ORCID
- Venue
- Journal of Internal Medicine
- Publisher
- Wiley
- Published
- 2013-08-10
- Volume
- 274
- Issue
- 4
- Pages
- 308-320
- ISSN
- 0954-6820 (ISSN-L)
- 1365-2796
- OpenAlex
- W2107605715
- Cited by
- 31
- References
- 77
- Citations by year
- 2025: 1 · 2023: 1 · 2020: 1 · 2019: 4 · 2018: 2 · 2017: 2 · 2016: 8 · 2015: 5 · 2014: 6 · 2013: 1
- FWCI
- 0.60
- Type
- journal article
- Language
- en
- Open access
- green
- kclpure.kcl.ac.uk
- Licence
- Locations
- Journal of Internal Medicine (publishedVersion)
- PubMed (publishedVersion)
- Research Portal (King's College London) (publishedVersion, other-oa)
- Funders
- Medical Research Council, UK (G0400061)
- EU Seventh Framework Programme (FP7/2007-2013)
- Topics
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Neuroscience and Neuropharmacology Research
- Keywords
- autism spectrum disorder, pharmacotherapy, personalized medicine, synaptic dysfunction, genetic variants, brain connectivity, neurobiology of autism, animal models, translational research, Innovative Medicines Initiative, clinical trial design
- MeSH terms
- Animals, Central Nervous System Agents, Child, Child Development Disorders, Pervasive, Child, Preschool, Disease Models, Animal, Genotype, Humans, Phenotype, Drug Design, Biomarkers, Translational Research, Biomedical
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