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Baraitser-Winter Syndrome 2

OMIM ID:

autosomal dominant?

Baraitser-Winter Syndrome 2

Alternate Names

BRWS2

Defective Genes

ACTG1

Clinical Characteristics

Ocular Features

Hypertelorism, high arched eyebrows, ptosis, and  colobomas occur in the majority of individuals.

Systemic Features

Short stature, postnatal microcephaly, lissencephaly, intellectual disability, seizures, and sensorineural hearing loss are common.

Genetics

Inheritance

This syndrome can be considered to be an autosomal dominant disorder secondary to heterozygous mutations in the ACTG1 gene (17q25.3).  However, all patients have been sporadic.

Mutations in ACTG1 are also responsible for autosomal dominant progressive nonsyndromic hearing loss.  

A similar but unique condition known as Baraitser-Winter syndrome 1 (243310) is caused by heterozygous mutations in the ACTB gene. 

Pedigree

Autosomal dominant

Autosomal dominant disorders require only one mutation for the disease to be expressed.  Since an affected parent has two chromosomes, only one of which has the mutant gene, parents can expect that half (50%) of their children will receive that one and inherit the disease.  It is common for individuals that inherit the mutation, however, to not have evidence of the disease (nonpenetrance).

Image
Sample pedigree of autosomal dominant inheritance

Autosomal dominant inheritance leads to a vertical pattern of transmission

Treatment & Management

There is no known treatment but special education, hearing devices, and physical therapy may be helpful.

Selected Resources

Publications

Displaying 1 - 3 of 3

Baraitser–Winter cerebrofrontofacial syndrome: delineation of the spectrum in 42 cases

PubMedID: 25052316

De novo mutations in the actin genes ACTB and ACTG1 cause Baraitser-Winter syndrome

PubMedID: 22366783

Iris coloboma, ptosis, hypertelorism, and mental retardation: a new syndrome

PubMedID: 3551890