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Cerebrofacioarticular syndrome

ORPHA:314679Malformation syndromeAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (2)

DCHS1
dachsous cadherin-related 1
Disease-causing germline mutation(s) in
OMIM: 603057
FAT4
FAT atypical cadherin 4
Disease-causing germline mutation(s) in
OMIM: 612411

Фенотипы (44)

Очень частый (80–99%)6
HP:0001263Global developmental delay
HP:0001999Abnormal facial shape
HP:0008551Microtia
HP:0008872Feeding difficulties in infancy
HP:0008947Floppy infant
HP:0012385Camptodactyly
Частый (30–79%)9
HP:0000089Renal hypoplasia
HP:0000239Large fontanelles
HP:0000347Micrognathia
HP:0000405Conductive hearing impairment
HP:0000938Osteopenia
HP:0001159Syndactyly
HP:0002342Intellectual disability, moderate
HP:0002778Abnormal trachea morphology
HP:0011471Gastrostomy tube feeding in infancy
Периодический (5–29%)29
HP:0000047Hypospadias
HP:0000160Narrow mouth
HP:0000252Microcephaly
HP:0000286Epicanthus
HP:0000316Hypertelorism
HP:0000327Hypoplasia of the maxilla
HP:0000431Wide nasal bridge
HP:0000581Blepharophimosis
HP:0001004Lymphedema
HP:0001251Ataxia
HP:0001274Agenesis of corpus callosum
HP:0001320Cerebellar vermis hypoplasia
HP:0001545Anteriorly placed anus
HP:0001627Abnormal heart morphology
HP:0001642Pulmonic stenosis
HP:0001762Talipes equinovarus
HP:0002025Anal stenosis
HP:0002079Hypoplasia of the corpus callosum
HP:0002119Ventriculomegaly
HP:0002282Gray matter heterotopia
HP:0002779Tracheomalacia
HP:0002825Caudal appendage
HP:0004322Short stature
HP:0006989Dysplastic corpus callosum
HP:0008197Absence of pubertal development
HP:0010864Intellectual disability, severe
HP:0040079Irregular dentition
HP:0100716Self-injurious behavior
HP:0200138Bilateral choanal atresia/stenosis

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы