Congenital fibrosis of the extraocular muscles

Gene: ZFHX4

Amber List (moderate evidence)

ZFHX4 (zinc finger homeobox 4)
EnsemblGeneIds (GRCh38): ENSG00000091656
EnsemblGeneIds (GRCh37): ENSG00000091656
OMIM: 606940, Gene2Phenotype
ZFHX4 is in 3 panels

2 reviews

Ida Ertmanska (Genomics England Curator)

Green List (high evidence)

Comment on list classification: There are more than 3 unrelated patients reported in literature with heterozygous ZFHX4 variants and isolated congenital ptosis. Hence, this gene can be promoted to Green at the next update.
Created: 22 Jul 2026, 1:47 p.m. | Last Modified: 22 Jul 2026, 1:47 p.m.
Panel Version: 2.5
PMID: 41524020 Zhang et al., 2026
Cohort of patients with isolated congenital ptosis. Seq method: WES. Three novel heterozygous variants in ZFHX4, c.5145C>A (p.N1715K), c.10382C>T (p.A3461V), and c.10795G>A (p.A3599T), were identified in three patients of Han Chinese origin.

PMID: 32962661 Adhikari et al., 2020
Report of a Nepalese proband with congenital ptosis and a c.12411G>T, p.Leu4137Phe variant in exon 12 of ZFHX4.
Same variant reported in a Japanese kindred with hereditary congenital blepharoptosis (PMID: 17987257, Nakashima et al., 2008).

PMID: 11935336 McMullan et al., 2002
Report of a patient with congenital bilateral isolated ptosis and a de novo balanced translocation 46,XY,t(1;8)(p34.3;q21.12). The 1p breakpoint does not disrupt a coding sequence, whereas the chromosome 8 breakpoint disrupts the ZFHX4 gene.

This gene is not yet associated with a disease entity in OMIM. ZFHX4 association with AD syndromic complex neurodevelopmental disorder was classified as Strong in ClinGen in December 2025 (Intellectual Disability and Autism GCEP).
Created: 22 Jul 2026, 1:41 p.m. | Last Modified: 22 Jul 2026, 1:46 p.m.
Panel Version: 2.4

Mode of inheritance
MONOALLELIC, autosomal or pseudoautosomal, NOT imprinted

Phenotypes
congenital ptosis, MONDO:0008340

Publications

Nicky Cronbach (UCL/Moorfields Eye Hospital)

Green List (high evidence)

Multiple unrelated cases of isolated congenital ptosis reported. Three unrelated Han-Chinese patients (PMID 41524020) and one Nepalese patient (32962661) with missense variants in ZFHX4, and one case (unspecified ethnicity) related to a translocation disrupting ZFHX4.
Sources: Literature
Created: 26 Jun 2026, 11:21 a.m.

Mode of inheritance
MONOALLELIC, autosomal or pseudoautosomal, NOT imprinted

Phenotypes
Isolated congenital ptosis

Publications

Details

Mode of Inheritance
MONOALLELIC, autosomal or pseudoautosomal, NOT imprinted
Sources
  • Expert Review Amber
Phenotypes
  • congenital ptosis, MONDO:0008340
Tags
Q3_26_NHS_review Q3_26_promote_green
OMIM
606940
Clinvar variants
Variants in ZFHX4
Penetrance
unknown
Publications
Panels with this gene

History Filter Activity

22 Jul 2026, Gel status: 2

Removed Tag, Added Tag

Ida Ertmanska (Genomics England Curator)

Tag Q3_26_expert_review was removed from gene: ZFHX4. Tag Q3_26_NHS_review tag was added to gene: ZFHX4.

22 Jul 2026, Gel status: 2

Added Tag, Added Tag

Ida Ertmanska (Genomics England Curator)

Tag Q3_26_expert_review tag was added to gene: ZFHX4. Tag Q3_26_promote_green tag was added to gene: ZFHX4.

22 Jul 2026, Gel status: 2

Set Phenotypes

Ida Ertmanska (Genomics England Curator)

Phenotypes for gene: ZFHX4 were changed from Isolated congenital ptosis to congenital ptosis, MONDO:0008340

22 Jul 2026, Gel status: 2

Set publications

Ida Ertmanska (Genomics England Curator)

Publications for gene: ZFHX4 were set to PMID: 41524020; 11935336; 32962661

22 Jul 2026, Gel status: 2

Entity classified by Genomics England curator

Ida Ertmanska (Genomics England Curator)

Gene: zfhx4 has been classified as Amber List (Moderate Evidence).

26 Jun 2026, Gel status: 0

Created, Added New Source, Set mode of inheritance, Set publications, Set Phenotypes, Set penetrance

Nicky Cronbach (UCL/Moorfields Eye Hospital)

gene: ZFHX4 was added gene: ZFHX4 was added to Congenital fibrosis of the extraocular muscles. Sources: Literature Mode of inheritance for gene: ZFHX4 was set to MONOALLELIC, autosomal or pseudoautosomal, NOT imprinted Publications for gene: ZFHX4 were set to PMID: 41524020; 11935336; 32962661 Phenotypes for gene: ZFHX4 were set to Isolated congenital ptosis Penetrance for gene: ZFHX4 were set to unknown Review for gene: ZFHX4 was set to GREEN