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Caudal regression syndrome

ORPHA:3027Malformation syndromeMultigenic/multifactorial, Not applicableAntenatal, Neonatal

Ассоциированные гены (2)

VANGL1
VANGL planar cell polarity protein 1
Major susceptibility factor in
OMIM: 610132
FUZ
fuzzy planar cell polarity protein
Disease-causing germline mutation(s) in
OMIM: 610622

Фенотипы (29)

Очень частый (80–99%)9
HP:0002607Bowel incontinence
HP:0002644Abnormality of pelvic girdle bone morphology
HP:0003199Decreased muscle mass
HP:0005640Abnormal vertebral segmentation and fusion
HP:0008479Hypoplastic vertebral bodies
HP:0008517Aplasia/Hypoplasia of the sacrum
HP:0009800Maternal diabetes
HP:0011867Abnormality of the wing of the ilium
HP:0100710Impulsivity
Частый (30–79%)11
HP:0000069Abnormality of the ureter
HP:0000073Ureteral duplication
HP:0000076Vesicoureteral reflux
HP:0000086Ectopic kidney
HP:0000104Renal agenesis
HP:0001315Reduced tendon reflexes
HP:0001387Joint stiffness
HP:0001762Talipes equinovarus
HP:0002023Anal atresia
HP:0002650Scoliosis
HP:0030680Abnormal cardiovascular system morphology
Периодический (5–29%)9
HP:0000028Cryptorchidism
HP:0000062Ambiguous genitalia
HP:0000083Renal insufficiency
HP:0000202Orofacial cleft
HP:0000822Hypertension
HP:0000921Missing ribs
HP:0002089Pulmonary hypoplasia
HP:0002139Arrhinencephaly
HP:0002308Chiari malformation

Эпидемиология (3)

Point prevalence
Unknown
Worldwide
Prevalence at birth
Unknown
Worldwide
Prevalence at birth
1-9 / 100 000
Australia

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы