Autistic Disorder
Recent clinical, regulatory, research and industry developments relating to this disease.
Synaptic signatures and disease vulnerabilities of layer 5 pyramidal neurons.
Nacc1 Mutation in Mice Models Rare Neurodevelopmental Disorder with Underlying Synaptic Dysfunction.
SynGAP regulates synaptic plasticity and cognition independently of its catalytic activity.
Autism Spectrum Disorder: Brain Areas Involved, Neurobiological Mechanisms, Diagnoses and Therapies.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 4 clinical trials expected to report results, the earliest in Q4 2026.
- Q4 2026An Exploratory Study of Spironolactone Tablets for the Treatment of Children With Gene Mutations Related to NCOR
- 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 Xanomeline and Trospium Chloride in Children and Adolescents With Psychiatric Disorders
- 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 MilestonesViewHide
- 2026-09-25An 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
- 2026-09-22A Phase 3 Multicenter, Open-label Study to Assess the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride in Children and Adolescents With Psychiatric DisordersResults expected Q1 2030
- 2026-05-18An Exploratory Study of Spironolactone Tablets for the Treatment of Children With Gene Mutations Related to NCORResults expected Q4 2026
- 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
- 2026-09-25ClinicalAn 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
- 2026-09-22ClinicalA Phase 3 Multicenter, Open-label Study to Assess the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride in Children and Adolescents With Psychiatric DisordersResults expected Q1 2030
- 2026-05-18ClinicalAn Exploratory Study of Spironolactone Tablets for the Treatment of Children With Gene Mutations Related to NCORResults expected Q4 2026
- 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
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Autistic Disorder15
- Autism Spectrum Disorder10
- Attention Deficit Disorder with Hyperactivity3
- Aggression1
- Alzheimer Disease1
- Brain1
- Brain Diseases1
- Cerebral Cortex1
Leading journals6
- Autism research : official journal of the International Society for Autism Research3
- Biology of sex differences2
- International journal of molecular sciences2
- Cell1
- Cerebral cortex (New York, N.Y. : 1991)1
- Frontiers in cellular and infection microbiology1
Leading researchers8
- Buxbaum JD3
- Cook EH2
- Devlin B2
- Durkin MS2
- Elsabbagh M2
- Fombonne E2
- Klei L2
- Reichert J2
Affiliations (unnormalised)6
- National Institute of Mental Health2
- University of California2
- Aarhus University1
- Agricultural Genetic Engineering Research Institute (AGERI)1
- Autism Research Centre1
- Bilkent University1
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
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.
Autistic disorder is a childhood-onset neurodevelopmental disorder marked by markedly impaired social interaction and communication together with a restricted repertoire of activities and interests. The clinical presentation varies widely with developmental level and age. The literature describes it as a heterogeneous, behaviorally defined condition.
The grounding supports a multifactorial neurodevelopmental basis rather than a single cause. Review abstracts emphasize genetic and epigenetic mutations, interactions with transcription factors, and chromatin remodeling processes in brain neurons. The literature also discusses biological sex as a factor that may modify prevalence estimates, but not as a proven cause.
The literature describes neurobiological alterations involving gene transcription, RNA and non-coding RNA translation, abnormal synaptic signaling pathways, and epigenetic post-translational mechanisms. One review also links ASD to effects on inhibitory and excitatory synaptic structures and to brain-area involvement. Resting-state EEG studies report differences across canonical frequency bands, consistent with altered brain network function.
The supplied grounding supports biological sex as a factor associated with differences in prevalence estimates. It also notes that sociodemographic status, ethnicity/race, and nativity have been examined as potential modifiers of prevalence, but does not establish them as risk factors. No other specific risk factors are supported by the grounding.
The grounding supports therapy at a general level but does not provide specific treatment recommendations. It indicates that ASD has no clear diagnostic markers and no specifically targeted medications, and that the literature discusses novel pharmacological therapy alongside multidisciplinary care. The treatment modality level supported here is behavioral and multidisciplinary management, with pharmacologic approaches remaining non-specific and investigational in the supplied material.
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.
A disorder beginning in childhood. It is marked by the presence of markedly abnormal or impaired development in social interaction and communication and a markedly restricted repertoire of activity and interest. Manifestations of the disorder vary greatly depending on the developmental level and chronological age of the individual. (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.