Disease Directory Williams Syndrome
Neurological

Williams Syndrome

Also known as: Williams-Beuren syndrome, WBS, 7q11.23 deletion syndrome, elastin deficiency

Prevalence

1-5 per 10,000 (Orphanet)

Onset

Childhood

Type

Genetic (chromosomal microdeletion, usually de novo)

Gene

ELN and adjacent genes at 7q11.23

About Williams Syndrome

Williams syndrome is caused by a heterozygous microdeletion of approximately 26-28 genes on chromosome 7q11.23, including the ELN gene encoding elastin. The characteristic phenotype includes elfin facial features, intellectual disability with a distinctive cognitive profile (strong verbal skills but weak visuospatial ability), hypersociability, cardiovascular disease (supravalvular aortic stenosis), hypercalcemia, and endocrine abnormalities.

Common Clinical Features

Supravalvular aortic stenosis Hypersociability Intellectual disability Elfin facial features Hypercalcemia Anxiety Renal abnormalities

Clinical Trial Eligibility Tips

What to know before applying to Williams Syndrome trials.

Chromosome 7q11.23 deletion confirmed by FISH, chromosomal microarray, or MLPA is required for trial eligibility

Cardiac evaluation including echocardiogram is required at baseline — SVAS severity affects eligibility and anesthetic risk

Cognitive profile testing (Wechsler scales, adaptive behavior) is a standard baseline measure

Calcium and vitamin D levels should be documented; hypercalcemia may be an exclusion criterion in some trials

Patient Resources

Patient Organization

Williams Syndrome Association

Visit website ↗

Natural History Registry

Williams Syndrome Association Research Registry

Join registry ↗

Orphanet

European reference resource for rare diseases (ORPHA:904)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

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Find recruiting Williams Syndrome trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Williams Syndrome. Updated daily.

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