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Weaver syndrome

ORPHA:3447Malformation syndromeAutosomal dominant, Not applicableAntenatal, Neonatal

Ассоциированные гены (3)

NSD1
nuclear receptor binding SET domain protein 1
Candidate gene tested in
OMIM: 606681
EZH2
enhancer of zeste 2 polycomb repressive complex 2 subunit
Disease-causing germline mutation(s) in
OMIM: 601573
SUZ12
SUZ12 polycomb repressive complex 2 subunit
Disease-causing germline mutation(s) (loss of function) in
OMIM: 606245

Фенотипы (42)

Очень частый (80–99%)22
HP:0005616Accelerated skeletal maturation
HP:0010300Abnormally low-pitched voice
HP:0000098Tall stature
HP:0000256Macrocephaly
HP:0000278Retrognathia
HP:0000316Hypertelorism
HP:0000337Broad forehead
HP:0000343Long philtrum
HP:0000347Micrognathia
HP:0000400Macrotia
HP:0000944Abnormal metaphysis morphology
HP:0001231Abnormal fingernail morphology
HP:0001249Intellectual disability
HP:0001257Spasticity
HP:0001263Global developmental delay
HP:0001276Hypertonia
HP:0001582Redundant skin
HP:0001609Hoarse voice
HP:0001800Hypoplastic toenails
HP:0001814Deep-set nails
HP:0001816Thin nail
HP:0000358Posteriorly rotated ears
Частый (30–79%)10
HP:0000023Inguinal hernia
HP:0000311Round face
HP:0001176Large hands
HP:0001387Joint stiffness
HP:0001769Broad foot
HP:0002002Deep philtrum
HP:0002213Fine hair
HP:0008872Feeding difficulties in infancy
HP:0011304Broad thumb
HP:0100490Camptodactyly of finger
Периодический (5–29%)10
HP:0000028Cryptorchidism
HP:0000494Downslanted palpebral fissures
HP:0001761Pes cavus
HP:0001762Talipes equinovarus
HP:0001852Sandal gap
HP:0002650Scoliosis
HP:0006101Finger syndactyly
HP:0008736Hypoplasia of penis
HP:0030680Abnormal cardiovascular system morphology
HP:0001382Joint hypermobility

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