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RERE-related neurodevelopmental syndrome

ORPHA:494344Malformation syndromeAutosomal dominantAntenatal, Neonatal

Ассоциированные гены (1)

RERE
arginine-glutamic acid dipeptide repeats
Disease-causing germline mutation(s) (loss of function) in
OMIM: 605226

Фенотипы (52)

Частый (30–79%)9
HP:0001627Abnormal heart morphology
HP:0008897Postnatal growth retardation
HP:0011968Feeding difficulties
HP:0410263Brain imaging abnormality
HP:0000478Abnormality of the eye
HP:0001249Intellectual disability
HP:0001252Hypotonia
HP:0001263Global developmental delay
HP:0001511Intrauterine growth retardation
Периодический (5–29%)43
HP:0000028Cryptorchidism
HP:0000047Hypospadias
HP:0000076Vesicoureteral reflux
HP:0000119Abnormality of the genitourinary system
HP:0000286Epicanthus
HP:0000347Micrognathia
HP:0000358Posteriorly rotated ears
HP:0000365Hearing impairment
HP:0000453Choanal atresia
HP:0000463Anteverted nares
HP:0000483Astigmatism
HP:0000486Strabismus
HP:0000508Ptosis
HP:0000545Myopia
HP:0000565Esotropia
HP:0000567Chorioretinal coloboma
HP:0000568Microphthalmia
HP:0000577Exotropia
HP:0000581Blepharophimosis
HP:0000612Iris coloboma
HP:0000648Optic atrophy
HP:0000659Peters anomaly
HP:0000708Atypical behavior
HP:0000729Autistic behavior
HP:0001250Seizure
HP:0001320Cerebellar vermis hypoplasia
HP:0001385Hip dysplasia
HP:0001629Ventricular septal defect
HP:0001999Abnormal facial shape
HP:0002007Frontal bossing
HP:0002015Dysphagia
HP:0002020Gastroesophageal reflux
HP:0002033Poor suck
HP:0002079Hypoplasia of the corpus callosum
HP:0002119Ventriculomegaly
HP:0002650Scoliosis
HP:0007018Attention deficit hyperactivity disorder
HP:0007305CNS demyelination
HP:0011229Broad eyebrow
HP:0012803Anisometropia
HP:0031910Abnormal cranial nerve physiology
HP:0100704Cerebral visual impairment
HP:0100716Self-injurious behavior

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