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Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome

ORPHA:294023DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

ADAM17
ADAM metallopeptidase domain 17
Disease-causing germline mutation(s) (loss of function) in
OMIM: 603639

Фенотипы (18)

Частый (30–79%)17
HP:0000498Blepharitis
HP:0001805Onychogryposis
HP:0003765Psoriasiform dermatitis
HP:0005406Recurrent bacterial skin infections
HP:0008396Chronic monilial nail infection
HP:0010783Erythema
HP:0011131Perianal rash
HP:0011228Horizontal eyebrow
HP:0011354Generalized abnormality of skin
HP:0012390Anal fissure
HP:0025085Bloody diarrhea
HP:0031123Recurrent gastroenteritis
HP:0040181Chapped lip
HP:0040189Scaling skin
HP:0100038Slow-growing scalp hair
HP:0200039Pustule
HP:0410017Otitis externa
Периодический (5–29%)1
HP:0001712Left ventricular hypertrophy

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