Trial reportThe Journal of pediatrics2014
Acquired autistic behaviors in children with mucopolysaccharidosis type IIIA.
Trial report in The Journal of pediatrics, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 1 synthesis or guideline pooled it, 52 citations in OpenAlex.
- Symptoms of Autism Spectrum Disorder (ASD) in Individuals with Mucopolysaccharide Disease Type III (Sanfilippo Syndrome): A Systematic Review.Journal of autism and developmental disorders · 2017Pooled it
- Anakinra in Sanfilippo syndrome: a phase 1/2 trial.Nature medicine · 2024Trial
- D1 dopamine receptor antagonists as a new therapeutic strategy to treat autistic-like behaviours in lysosomal storage disorders.Molecular psychiatry · 2025Article
- Neurocognitive testing in a murine model of mucopolysaccharidosis type IIIA.Molecular genetics and metabolism reports · 2023Article
- Sanfilippo syndrome: consensus guidelines for clinical care.Orphanet journal of rare diseases · 2022Review
- Genetic mouse models of autism spectrum disorder present subtle heterogenous cardiac abnormalities.Autism research : official journal of the International Society for Autism Research · 2022Article
- An ethical analysis of divergent clinical approaches to the application of genetic testing for autism and schizophrenia.Human genetics · 2022Article
- Music therapy and Sanfilippo syndrome: an analysis of psychological and physiological variables of three case studies.Orphanet journal of rare diseases · 2021Article
- Altered heparan sulfate metabolism during development triggers dopamine-dependent autistic-behaviours in models of lysosomal storage disorders.Nature communications · 2021Article
- The Development of the Mesoprefrontal Dopaminergic System in Health and Disease.Frontiers in neural circuits · 2021Review
- Challenging behavior in mucopolysaccharidoses types I-III and day-to-day coping strategies: a cross sectional explorative study.Orphanet journal of rare diseases · 2020Review
- Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease.Journal of clinical medicine · 2020Review
- Neurological Diseases With Autism Spectrum Disorder: Role of ASD Risk Genes.Frontiers in neuroscience · 2019Article
- A novel mutation of SGSH and clinical features analysis of mucopolysaccharidosis type IIIA.Medicine · 2018Article
- Neurobehavioral phenotypes of neuronopathic mucopolysaccharidoses.Italian journal of pediatrics · 2018Review
- MPS-IIIA mice acquire autistic behaviours with age.Journal of inherited metabolic disease · 2018Article
- Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.Orphanet journal of rare diseases · 2017Article
- Identification of the Genetic Cause for Childhood Disintegrative Disorder by Whole-Exome Sequencing.Neuroscience bulletin · 2017Article
- Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice.Behavioural brain research · 2016Article
- Glycan susceptibility factors in autism spectrum disorders.Molecular aspects of medicine · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
objectivesTo assess autism spectrum disorder (ASD) behaviors in children with mucopolysaccharidosis type IIIA (MPS IIIA) using a standard measure, understand the behavioral evolution of the disease, and provide specific guidelines for diagnosis. STUDY
designChildren (n = 21) with documented enzyme deficiency and SGSH gene mutations, cognitive age-equivalent >12 months, and early onset were administered the Autism Diagnostic Observation Schedule (ADOS) (module 1) and Bayley Scales of Infant Development-Third Edition. ADOS Social Affect and Restricted Repetitive Behavior total scores, as well as Bayley Scales of Infant Development-Third Edition cognitive age-equivalent scores, are reported using descriptive statistics and graphic presentations.
resultsThirteen of the 21 children evaluated met the ADOS criteria for ASD/autism. ADOS score was strongly associated with age; all 11 children aged >46 months met the criteria, compared with only 2 of 10 aged <46 months. Social and affective abnormalities were most frequent; restricted interests and repetitive behaviors were largely absent. Lack of cognitive growth paralleled ADOS score.
conclusionAn increased incidence of ASD-like social behaviors was seen at age 3-4 years in children with early-onset MPS IIIA. Although more frequent in the severely impaired children, ASD-like behaviors were observed across the entire range of cognitive impairment. Clinicians must be aware that when a child acquires ASD-like behaviors, MPS IIIA should be included in the differential diagnosis.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.