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Male emopamil-binding protein disorder with neurological defect

MedGen UID:
1680758
Concept ID:
C5190584
Disease or Syndrome
Synonyms: Male EBP (emopamil-binding protein) disorder with neurological defect; MEND (male emopamil-binding protein disorder with neurological defect) syndrome
SNOMED CT: Male emopamil-binding protein disorder with neurological defect (782739000); Male EBP (emopamil-binding protein) disorder with neurological defect (782739000); MEND (male emopamil-binding protein disorder with neurological defect) syndrome (782739000)

Definition

A rare genetic syndromic sterol biosynthesis disorder affecting males. The disease has characteristics of skin manifestations including collodion membrane, ichthyosis and patchy hypopigmented lesions associated with severe neurological involvement (for example intellectual disability, delayed psychomotor development, seizures, hydrocephalus, cerebellar/corpus callosum hypoplasia, Dandy-Walker malformation, hypotonia) and craniofacial dysmorphism (large anterior fontanelle, telecanthus, hypertelorism, microphthalmia, prominent nasal bridge, low-set ears, micrognathia, cleft palate). Toe syndactyly, polydactyly and kyphosis as well as ophthalmic, cardiac and urogenital anomalies may also be associated. There is evidence the disease is caused by hemizygous mutation in the EBP gene on chromosome Xp11. [from SNOMEDCT_US]

Recent clinical studies

Diagnosis

Arnold AW, Bruckner-Tuderman L, Has C, Happle R
Br J Dermatol 2012 Jun;166(6):1309-13. Epub 2012 Mar 27 doi: 10.1111/j.1365-2133.2012.10808.x. PMID: 22229330

Prognosis

Barboza-Cerda MC, Barboza-Quintana O, Martínez-Aldape G, Garza-Guajardo R, Déctor MA
Mol Genet Genomic Med 2019 Sep;7(9):e931. Epub 2019 Aug 8 doi: 10.1002/mgg3.931. PMID: 31397093Free PMC Article
Arnold AW, Bruckner-Tuderman L, Has C, Happle R
Br J Dermatol 2012 Jun;166(6):1309-13. Epub 2012 Mar 27 doi: 10.1111/j.1365-2133.2012.10808.x. PMID: 22229330

Clinical prediction guides

Barboza-Cerda MC, Barboza-Quintana O, Martínez-Aldape G, Garza-Guajardo R, Déctor MA
Mol Genet Genomic Med 2019 Sep;7(9):e931. Epub 2019 Aug 8 doi: 10.1002/mgg3.931. PMID: 31397093Free PMC Article

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