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Otopalatodigital syndrome type 1

ORPHA:90650Malformation syndromeX-linked dominantAntenatal, Infancy, Neonatal

Ассоциированные гены (1)

FLNA
filamin A
Disease-causing germline mutation(s) (gain of function) in
OMIM: 300017

Фенотипы (27)

Очень частый (80–99%)14
HP:0000175Cleft palate
HP:0000316Hypertelorism
HP:0000336Prominent supraorbital ridges
HP:0000365Hearing impairment
HP:0000431Wide nasal bridge
HP:0000494Downslanted palpebral fissures
HP:0000674Anodontia
HP:0000677Oligodontia
HP:0001256Intellectual disability, mild
HP:0001376Limitation of joint mobility
HP:0001852Sandal gap
HP:0002652Skeletal dysplasia
HP:0005280Depressed nasal bridge
HP:0010109Short hallux
Частый (30–79%)10
HP:0002684Thickened calvaria
HP:0002738Hypoplastic frontal sinuses
HP:0003042Elbow dislocation
HP:0004279Short palm
HP:0005916Abnormal metacarpal morphology
HP:0006487Bowing of the long bones
HP:0009623Proximal placement of thumb
HP:0009778Short thumb
HP:0009882Short distal phalanx of finger
HP:0011001Increased bone mineral density
Периодический (5–29%)3
HP:0001850Abnormality of the tarsal bones
HP:0005048Synostosis of carpal bones
HP:0005640Abnormal vertebral segmentation and fusion

Эпидемиология (1)

Point prevalence
1-9 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы