Disease Directory Wolf-Hirschhorn Syndrome
Neurological

Wolf-Hirschhorn Syndrome

Also known as: WHS, 4p deletion syndrome, 4p16.3 monosomy, Pitt-Rogers-Danks syndrome

Prevalence

1-9 per 100,000 (Orphanet)

Onset

Neonatal, Infantile

Type

Genetic (chromosomal deletion, usually de novo)

Gene

WHSC1 (NSD2), FGFRL1 and adjacent genes at 4p16.3

About Wolf-Hirschhorn Syndrome

Wolf-Hirschhorn syndrome is caused by partial deletion of the short arm of chromosome 4 (4p16.3), resulting in haploinsufficiency of multiple genes including WHSC1 (NSD2) and FGFRL1. The characteristic 'Greek warrior helmet' facial appearance, intrauterine growth restriction, intellectual disability, seizures, and midline defects (cleft palate, heart defects) define the syndrome. Severity correlates with deletion size. Seizures are present in ~75% and may respond to valproate.

Common Clinical Features

Greek warrior helmet facial appearance Intrauterine growth restriction Intellectual disability Seizures Hypotonia Midline defects (heart, palate) Short stature

Clinical Trial Eligibility Tips

What to know before applying to Wolf-Hirschhorn Syndrome trials.

Chromosomal microarray (CMA) documenting 4p16.3 deletion size is required — deletion size influences phenotype and trial stratification

Seizure history, type, and current antiseizure medication regimen are required baseline information

Growth parameters (height, weight, head circumference) and cardiac evaluation are standard baseline assessments

Natural history and observational studies are the primary research option; contact Chromosome 4p Support Group for trial matching

Patient Resources

Patient Organization

Wolf-Hirschhorn Syndrome Support Group

Visit website ↗

Orphanet

European reference resource for rare diseases (ORPHA:280)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Wolf-Hirschhorn Syndrome trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Wolf-Hirschhorn Syndrome. Updated daily.

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