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Bowen-Conradi syndrome

ORPHA:1270Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

EMG1
EMG1 N1-specific pseudouridine methyltransferase
Disease-causing germline mutation(s) in
OMIM: 611531

Фенотипы (20)

Очень частый (80–99%)10
HP:0000252Microcephaly
HP:0000340Sloping forehead
HP:0000347Micrognathia
HP:0000448Prominent nose
HP:0001387Joint stiffness
HP:0001522Death in infancy
HP:0004322Short stature
HP:0008850Severe postnatal growth retardation
HP:0008872Feeding difficulties in infancy
HP:0011344Severe global developmental delay
Частый (30–79%)5
HP:0000028Cryptorchidism
HP:0001838Rocker bottom foot
HP:0004209Clinodactyly of the 5th finger
HP:0008846Severe intrauterine growth retardation
HP:0100490Camptodactyly of finger
Периодический (5–29%)5
HP:0000202Orofacial cleft
HP:0001250Seizure
HP:0002101Abnormal lung lobation
HP:0002119Ventriculomegaly
HP:0030680Abnormal cardiovascular system morphology

Эпидемиология (3)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide
Prevalence at birth
>1 / 1000
Specific population

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы