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Pelviscapular dysplasia

ORPHA:93333Malformation syndromeAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

TBX15
T-box transcription factor 15
Disease-causing germline mutation(s) (loss of function) in
OMIM: 604127

Фенотипы (24)

Облигатный (100%)11
HP:0000256Macrocephaly
HP:0000470Short neck
HP:0000882Hypoplastic scapulae
HP:0000946Hypoplastic ilia
HP:0001156Brachydactyly
HP:0001374Congenital hip dislocation
HP:0002987Elbow flexion contracture
HP:0003041Humeroradial synostosis
HP:0003097Short femur
HP:0003943Abnormality of the joint spaces of the elbow
HP:0004987Mesomelic leg shortening
Очень частый (80–99%)3
HP:0000369Low-set ears
HP:0002693Abnormality of the skull base
HP:0004322Short stature
Частый (30–79%)10
HP:0000316Hypertelorism
HP:0000365Hearing impairment
HP:0000377Abnormal pinna morphology
HP:0000402Stenosis of the external auditory canal
HP:0000486Strabismus
HP:0000490Deeply set eye
HP:0000581Blepharophimosis
HP:0002007Frontal bossing
HP:0002162Low posterior hairline
HP:0005989Redundant neck skin

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