Autism Spectrum Disorder
Recent clinical, regulatory, research and industry developments relating to this disease.
Population-Based Risk of Psychiatric Disorders Associated With Recurrent Copy Number Variants.
Prenatal environmental risk factors for autism spectrum disorder and their potential mechanisms.
Key Synaptic Pathology in Autism Spectrum Disorder: Genetic Mechanisms and Recent Advances.
Multivariate brain-behaviour associations in psychiatric disorders.
Transcriptomic sex differences in postmortem brain samples from patients with psychiatric disorders.
Cross-ancestry atlas of gene, isoform, and splicing regulation in the developing human brain.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 5 clinical trials expected to report results, the earliest in Q1 2027.
- 1 industry development reported.
- Q1 2027Comparative Effectiveness of Pharmacologic Management of ADHD in Children and Youth With Autism Spectrum Disorder
- Q2 2027A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of Age
- Q1 2030A Pharmaco-Imaging Trial of Mixed Amphetamine Salts for the Treatment of ADHD in Intellectually-intact Youth With Autism Spectrum Disorder
- Q1 2030A Phase 3 Multicenter, Open-label Study to Assess the Long-term Safety and Tolerability of KarXT in Adolescents (13 to 17 Years of Age) With Schizophrenia and KarXT+KarX-EC in Children and Adolescents (5 to 17 Years of Age) With Irritability Associated With Autism Spectrum Disorder
- Q2 2032An Open-label, Multicenter Trial to Assess the Safety and Tolerability of Lumateperone in the Treatment of Pediatric Patients With Schizophrenia, Bipolar Disorder or Autism Spectrum Disorder
Clinical MilestonesView all 10Hide
- 2026-07-31An Open-label, Multicenter Trial to Assess the Safety and Tolerability of Lumateperone in the Treatment of Pediatric Patients With Schizophrenia, Bipolar Disorder or Autism Spectrum DisorderResults expected Q2 2032
- 2026-07-07A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of AgeResults expected Q2 2027
- 2026-06-23A Phase 3 Multicenter, Open-label Study to Assess the Long-term Safety and Tolerability of KarXT in Adolescents (13 to 17 Years of Age) With Schizophrenia and KarXT+KarX-EC in Children and Adolescents (5 to 17 Years of Age) With Irritability Associated With Autism Spectrum DisorderResults expected Q1 2030
- 2026-03-06A Pharmaco-Imaging Trial of Mixed Amphetamine Salts for the Treatment of ADHD in Intellectually-intact Youth With Autism Spectrum DisorderResults expected Q1 2030
- 2025-10-29Comparative Effectiveness of Pharmacologic Management of ADHD in Children and Youth With Autism Spectrum DisorderResults expected Q1 2027
- 2025-12-31Biomarker-Driven Pharmacological Treatment for Autism Spectrum DisorderCompleted
- 2025-12-01Pragmatic Trial Comparing Weight Gain in Children With Autism Taking Risperidone Versus AripiprazoleCompleted
- 2025-10-06A 52-Week, Multicenter, Open-Label, Flexible-dose Study to Evaluate the Long-term Safety and Tolerability of Cariprazine in the Treatment of Pediatric Subjects With Schizophrenia, Bipolar I Disorder, or Autism Spectrum DisorderCompleted
Industry & MarketViewHide
- 2026-06-17Cortical development dynamics across autism spectrum disorder mouse modelsIndustry · Nature News
- 2026-07-31ClinicalAn Open-label, Multicenter Trial to Assess the Safety and Tolerability of Lumateperone in the Treatment of Pediatric Patients With Schizophrenia, Bipolar Disorder or Autism Spectrum DisorderResults expected Q2 2032
- 2026-07-07ClinicalA Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of AgeResults expected Q2 2027
- 2026-06-23ClinicalA Phase 3 Multicenter, Open-label Study to Assess the Long-term Safety and Tolerability of KarXT in Adolescents (13 to 17 Years of Age) With Schizophrenia and KarXT+KarX-EC in Children and Adolescents (5 to 17 Years of Age) With Irritability Associated With Autism Spectrum DisorderResults expected Q1 2030
- 2026-06-17IndustryCortical development dynamics across autism spectrum disorder mouse modelsIndustry · Nature News
- 2026-03-06ClinicalA Pharmaco-Imaging Trial of Mixed Amphetamine Salts for the Treatment of ADHD in Intellectually-intact Youth With Autism Spectrum DisorderResults expected Q1 2030
- 2025-12-31ClinicalBiomarker-Driven Pharmacological Treatment for Autism Spectrum DisorderCompleted
- 2025-12-01ClinicalPragmatic Trial Comparing Weight Gain in Children With Autism Taking Risperidone Versus AripiprazoleCompleted
- 2025-11-25ClinicalMemantine for the Treatment of Social Deficits in Youth With Disorders of Impaired Social Interactions: A Randomized-controlled TrialTerminated
- 2025-10-29ClinicalComparative Effectiveness of Pharmacologic Management of ADHD in Children and Youth With Autism Spectrum DisorderResults expected Q1 2027
- 2025-10-06ClinicalA 52-Week, Multicenter, Open-Label, Flexible-dose Study to Evaluate the Long-term Safety and Tolerability of Cariprazine in the Treatment of Pediatric Subjects With Schizophrenia, Bipolar I Disorder, or Autism Spectrum DisorderCompleted
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — Slenyto is indicated for: treatment of insomnia in children and adolescents aged 2-18 ye… (2018)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Autism Spectrum Disorder65
- Attention Deficit Disorder with Hyperactivity11
- Gastrointestinal Microbiome11
- Mental Disorders11
- Autistic Disorder10
- Major Depressive Disorder7
- Bipolar Disorder6
- Neurodevelopmental Disorders6
Leading journals6
- Molecular psychiatry9
- International journal of molecular sciences8
- Morbidity and mortality weekly report. Surveillance summaries (Washington, D.C. : 2002)6
- Translational psychiatry6
- Molecular autism5
- BMC medicine3
Leading researchers8
- Durkin MS7
- Fitzgerald RT6
- Pettygrove S6
- Zahorodny W6
- Hall-Lande J5
- Lopez M5
- Maenner MJ5
- Shenouda J5
Affiliations (unnormalised)6
- School of Medicine5
- Donders Institute for Brain4
- Imaging Genetics Center4
- Institute of Psychiatry4
- Karolinska Institutet4
- National Institute of Mental Health4
Disease biology
Key proteins & gene products studied in this disease. Number shows shared papers.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Autism spectrum disorder is a wide continuum of cognitive and neurobehavioral conditions defined by core impairments in socialization, verbal and nonverbal communication, and restricted, repetitive patterns of behavior. The literature grounding also treats it as a neurodevelopmental disorder with substantial genetic and biological heterogeneity.
The supplied grounding supports a genetic contribution, including associations from genome-wide studies and single-nucleotide polymorphism analyses. It also supports investigation of microbiome-related and brain-gut axis factors, but does not establish a single cause.
The literature grounding points to altered neuronal plasticity, signal transduction, transcriptome and gene regulatory network changes, and involvement of proteins such as trkB, cytokines, tumor necrosis factor-alpha, interleukin-4, and membrane proteins. It also supports a role for gut microbiota and microbiota-derived metabolites in bidirectional brain-gut communication, with immunological, neuronal, and endocrine pathways implicated.
The supplied grounding supports biological sex, sociodemographic status, ethnicity/race, and nativity as factors examined in relation to prevalence variation, but not as established causal risk factors. It also includes genetic variation and microbiome-related factors as areas of study, without defining them as confirmed risks.
The grounding supports therapy and drug therapy as literature topics, but it does not provide specific treatment standards or modalities for autism spectrum disorder. No supported standard-of-care treatment category can be stated from the supplied material alone.
AI-generated summary grounded in MeSH and 6 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Wide continuum of associated cognitive and neurobehavioral disorders, including, but not limited to, three core-defining features: impairments in socialization, impairments in verbal and nonverbal communication, and restricted and repetitive patterns of behaviors. (from DSM-V)
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.