Miller syndrome, also called Genée-Wiedemann syndrome, Wildervanck-Smith syndrome or postaxial acrofacial dysostosis, is an extremely rare genetic condition affecting the face, limbs and eyes. It is caused by a recessive mutation in the DHODH gene on chromosome 16, which encodes an enzyme needed for building pyrimidines in cells. Features include a very small lower jaw, cleft lip or palate, missing or underdeveloped bones on the outer side of the hands and feet, eyelid coloboma, downslanting eyelid openings, malformed ears and extra nipples. Some people also have vertebral and rib defects, heart defects and lung disease from chronic infection. The condition was first described in 1969 by Ekkart Genée, who took it for an extreme form of Treacher Collins syndrome, and Hans-Rudolf Wiedemann described it as a separate entity in 1975. A family with Miller syndrome was the first to have its whole genome sequenced.
Facts
Classification
ICD-10 ChapterXVII: Congenital Malformations, Deformations and Chromosomal Abnormalities 1 Communicable or Non-communicable Transmission Route First Described Prevalence Band Causative Agent
Causative Agent (category) Sources
1. Orphadata (Orphanet): Orphadata cross-references for ORPHA:246 Miller syndrome
Orphadata (Orphanet)ICD-10 cross-referenceQuote, ICD-10 cross-reference
"Reference": "Q75.4", "Source": "ICD-10"
View the Source 2. Miller syndrome (Wikipedia)
Wikipedia, Lead
It is caused by a mutation in the DHODH gene
Wikipedia, Lead [disease-category]
is an extremely rare genetic condition
Wikipedia, History
This condition was first described in 1969 by Genée
View the Source3. MedlinePlus Genetics (U.S. National Library of Medicine): Miller syndrome - MedlinePlus Genetics
MedlinePlus Genetics (U.S. National Library of Medicine)Description and InheritanceQuote, Description and Inheritance
Though dihydroorotate dehydrogenase appears to be active in the limb buds during the early stages of development, it is not clear how variants in the DHODH gene cause the specific bone abnormalities seen in people with Miller syndrome.
View the Source 4. Orphadata (Orphanet): Orphadata epidemiology for ORPHA:246 Postaxial acrofacial dysostosis
Orphadata (Orphanet)Prevalence, WorldwideQuote, Prevalence, Worldwide
"PrevalenceClass": "\u003c1 / 1 000 000", "PrevalenceGeographic": "Worldwide"
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