← Назад

Epidermolysis bullosa simplex with pyloric atresia

ORPHA:158684DiseaseAutosomal recessiveNeonatal

Ассоциированные гены (2)

PLEC
plectin
Disease-causing germline mutation(s) in
OMIM: 601282
ITGB4
integrin subunit beta 4
Disease-causing germline mutation(s) in
OMIM: 147557

Фенотипы (29)

Частый (30–79%)12
HP:0000079Abnormality of the urinary system
HP:0001030Fragile skin
HP:0001057Aplasia cutis congenita
HP:0002013Vomiting
HP:0003236Elevated circulating creatine kinase concentration
HP:0003270Abdominal distention
HP:0003341Junctional split
HP:0004399Congenital pyloric atresia
HP:0007585Skin fragility with non-scarring blistering
HP:0008066Abnormal blistering of the skin
HP:0200041Skin erosion
HP:0200097Oral mucosal blisters
Периодический (5–29%)17
HP:0000070Ureterocele
HP:0000075Renal duplication
HP:0000096Glomerulosclerosis
HP:0000110Renal dysplasia
HP:0000126Hydronephrosis
HP:0000795Abnormality of the urethra
HP:0001056Milia
HP:0001075Atrophic scars
HP:0001371Flexion contracture
HP:0001561Polyhydramnios
HP:0003560Muscular dystrophy
HP:0004552Scarring alopecia of scalp
HP:0007385Aplasia cutis congenita of scalp
HP:0007589Aplasia cutis congenita on trunk or limbs
HP:0008404Nail dystrophy
HP:0008551Microtia
HP:0010477Aplasia of the bladder

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы