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FRAXA

Chr XXLDXLRX-linked

fragile site, folic acid type, rare, fra(X)(q27.3) A

Also known as: FMR1

The FRAXA locus contains a CGG trinucleotide repeat in the 5' regulatory region of the FMR1 gene that encodes fragile X messenger ribonucleoprotein 1. Expansions of this repeat cause fragile X syndrome (the most common inherited cause of intellectual disability and autism spectrum disorder) when fully expanded beyond 200 repeats, and fragile X tremor/ataxia syndrome or premature ovarian insufficiency with intermediate expansions of 55-200 repeats. The condition follows X-linked inheritance with reduced penetrance in females due to X-inactivation, and repeat expansions show a bias toward further expansion during maternal transmission.

OMIMResearchSummary from RefSeq, OMIM
MultiplemechanismXLD/XLR/X-linked5 OMIM phenotypes
Clinical SummaryFRAXA
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Clinical Trials
9 active or recruiting trials — potential therapeutic options may be available
Some data sources returned errors (2)

ensembl: Error: Ensembl fetch failed: 400 for https://rest.ensembl.org/lookup/symbol/homo_sapiens/FRAXA?content-type=application/json&expand=1

gnomad: Error: Gene not found

Population Genetics & Constraint

Constraint data not available from gnomAD.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

FRAXA · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Specific Language and Learning Disorders (SLLD)

Study of the Value of Trio Exome Sequencing in the Etiological Assessment of Specific Non-syndromic Language and Learning Disorders

RECRUITING
NCT05939739Phase NACentre Hospitalier Universitaire DijonStarted 2023-08-07
Blood samplesConsultation for results deliveryStudy Humanities and Social Sciences
Fragile X Syndrome (FXS)Autism Spectrum Disorder

Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder

RECRUITING
NCT06677866Phase NABambino Gesù Hospital and Research InstituteStarted 2022-09-01
Cognitive behavioral therapyWaiting List
Primary Ovarian Insufficiency

Investigation of Copy Number Variations and Genetic Variants in POI

RECRUITING
NCT05327283Ospedale Policlinico San MartinoStarted 2012-01-31
Fragile X Syndrome (FXS)Creatine Transporter Deficiency

Optical Imaging in X-linked Disorders.

RECRUITING
NCT06868979Phase NAHospices Civils de LyonStarted 2026-03-30
Clinical assessmentParental questionnairesCognitive assessment
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, IncludingSickle Cell DiseaseCystic Fibrosis

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

RECRUITING
NCT06147414Assistance Publique - Hôpitaux de ParisStarted 2024-10-23
Blood sample
FMR1 Gene Premutation

Assessment of Ovarian Reserve in Patients With Fragile X Premutation

RECRUITING
NCT07039734Assistance Publique - Hôpitaux de ParisStarted 2025-07-04
collection of data from medical records
Fragile X Syndrome

Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals with Fragile-X Syndrome

NOT YET RECRUITING
NCT06261502Phase PHASE2Université de SherbrookeStarted 2025-05-01
CBD Oral SolutionPlacebo
Fragile X SyndromeAutism Spectrum DisorderAutistic Disorder

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders

RECRUITING
NCT06227780Phase NAChildren's Hospital Medical Center, CincinnatiStarted 2023-05-24
Alpha Auditory EntrainmentSham
Fragile X Syndrome

Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome

RECRUITING
NCT06279858Phase NASpecila hospital for cerebral palsy and developmental neurologyStarted 2024-01-01
Probiotics
Clinical Literature
Open Research Assistant →
Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC