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Catel-Manzke syndrome

ORPHA:1388Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

TGDS
TDP-glucose 4,6-dehydratase
Disease-causing germline mutation(s) in
OMIM: 616146

Фенотипы (25)

Очень частый (80–99%)7
HP:0000162Glossoptosis
HP:0000175Cleft palate
HP:0000272Malar flattening
HP:0000347Micrognathia
HP:0001508Failure to thrive
HP:0004209Clinodactyly of the 5th finger
HP:0005930Abnormality of epiphysis morphology
Частый (30–79%)9
HP:0000293Full cheeks
HP:0000389Chronic otitis media
HP:0001387Joint stiffness
HP:0001629Ventricular septal defect
HP:0002553Highly arched eyebrow
HP:0002650Scoliosis
HP:0004322Short stature
HP:0030680Abnormal cardiovascular system morphology
HP:0000358Posteriorly rotated ears
Периодический (5–29%)9
HP:0100490Camptodactyly of finger
HP:0000316Hypertelorism
HP:0000767Pectus excavatum
HP:0001631Atrial septal defect
HP:0002119Ventriculomegaly
HP:0009467Radial deviation of the 2nd finger
HP:0010285Oral synechia
HP:0010508Metatarsus valgus
HP:0001382Joint hypermobility

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