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Developmental and speech delay due to SOX5 deficiency

ORPHA:313892Clinical subtypeAutosomal dominant, Not applicableInfancy, Neonatal

Ассоциированные гены (1)

SOX5
SRY-box transcription factor 5
Disease-causing germline mutation(s) in
OMIM: 604975

Фенотипы (31)

Облигатный (100%)2
HP:0001249Intellectual disability
HP:0001270Motor delay
Очень частый (80–99%)2
HP:0000750Delayed speech and language development
HP:0430028Hyperplasia of the maxilla
Частый (30–79%)16
HP:0000189Narrow palate
HP:0000486Strabismus
HP:0000545Myopia
HP:0000577Exotropia
HP:0000648Optic atrophy
HP:0000678Dental crowding
HP:0000739Anxiety
HP:0000768Pectus carinatum
HP:0001252Hypotonia
HP:0002007Frontal bossing
HP:0002711Exaggerated median tongue furrow
HP:0002938Lumbar hyperlordosis
HP:0002948Vertebral fusion
HP:00046912-3 toe syndactyly
HP:0005659Thoracic kyphoscoliosis
HP:0012443Abnormality of brain morphology
Периодический (5–29%)11
HP:0000078Abnormality of the genital system
HP:0000718Aggressive behavior
HP:0000733Abnormal repetitive mannerisms
HP:0001250Seizure
HP:0001653Mitral regurgitation
HP:0002020Gastroesophageal reflux
HP:0002650Scoliosis
HP:0003316Butterfly vertebrae
HP:0007018Attention deficit hyperactivity disorder
HP:0011968Feeding difficulties
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