Neurological

Mowat-Wilson Syndrome

Also known as MWS, ZEB2 haploinsufficiency, Hirschsprung disease with intellectual disability

Mowat-Wilson syndrome is caused by de novo loss-of-function mutations or deletions of ZEB2, encoding Zinc finger E-box binding homeobox 2, a transcriptional repressor critical for neural crest development. Clinical features include distinct

ORPHA:261552 ↗Gene ZEB2Prevalence 1-9 per 100,000 (Orphanet)Onset Infantile, ChildhoodAutosomal dominant genetic (de novo in most cases)

0

studies recruiting now

as of 7 Sept 2026

1

studies registered in total

as of 7 Sept 2026

0

countries with a recruiting site

as of 7 Sept 2026

None

recruiting study posted to date

among recruiting studies

Recruiting trials

No recruiting trial found right now.

1 study is registered for Mowat-Wilson Syndrome, but none was recruiting as of 7 Sept 2026. Here is what is still worth doing.

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Patient organisations

Mowat-Wilson Syndrome FoundationPatient association
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Registry: Mowat-Wilson Syndrome International Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

About Mowat-Wilson Syndrome

Mowat-Wilson syndrome is caused by de novo loss-of-function mutations or deletions of ZEB2, encoding Zinc finger E-box binding homeobox 2, a transcriptional repressor critical for neural crest development. Clinical features include distinctive facial appearance (deeply set eyes, prominent columella), moderate-to-severe intellectual disability, absent or severely limited speech, Hirschsprung disease (in ~50%), epilepsy, and structural brain abnormalities including corpus callosum hypoplasia.

Common clinical features

Moderate-to-severe intellectual disabilityAbsent or limited speechHirschsprung diseaseEpilepsyDistinctive facial featuresCorpus callosum hypoplasiaShort stature

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Before you apply

Things trial teams commonly ask about for Mowat-Wilson Syndrome. Not eligibility rules; those are set by each study.

  • ZEB2 pathogenic variant confirmed by sequence analysis and/or deletion/duplication testing is required for trial eligibility
  • Hirschsprung disease history and surgical intervention status should be documented — GI complications may affect trial eligibility
  • Seizure type and current antiseizure medication regimen must be stable for a defined period before enrollment
  • Adaptive behavior and communication assessments are the primary outcome measures in behavioral and pharmacological trials

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).