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Junctional epidermolysis bullosa with pyloric atresia

ORPHA:79403DiseaseAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (2)

ITGA6
integrin subunit alpha 6
Disease-causing germline mutation(s) in
OMIM: 147556
ITGB4
integrin subunit beta 4
Disease-causing germline mutation(s) in
OMIM: 147557

Фенотипы (21)

Очень частый (80–99%)6
HP:0002017Nausea and vomiting
HP:0003270Abdominal distention
HP:0004399Congenital pyloric atresia
HP:0008066Abnormal blistering of the skin
HP:0011100Intestinal atresia
HP:0200097Oral mucosal blisters
Частый (30–79%)11
HP:0000070Ureterocele
HP:0000075Renal duplication
HP:0000110Renal dysplasia
HP:0000126Hydronephrosis
HP:0000790Hematuria
HP:0001561Polyhydramnios
HP:0001581Recurrent skin infections
HP:0006297Enamel hypoplasia
HP:0010477Aplasia of the bladder
HP:0012227Urethral stricture
HP:0100577Urinary bladder inflammation
Периодический (5–29%)4
HP:0000656Ectropion
HP:0001057Aplasia cutis congenita
HP:0001059Pterygium
HP:0008404Nail dystrophy

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