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Self-improving dystrophic epidermolysis bullosa

ORPHA:79411DiseaseAutosomal dominant, Autosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

COL7A1
collagen type VII alpha 1 chain
Disease-causing germline mutation(s) (loss of function) in
OMIM: 120120

Фенотипы (27)

Очень частый (80–99%)1
HP:0011354Generalized abnormality of skin
Частый (30–79%)8
HP:0001030Fragile skin
HP:0001056Milia
HP:0001075Atrophic scars
HP:0001798Anonychia
HP:0008066Abnormal blistering of the skin
HP:0008404Nail dystrophy
HP:0009723Abnormality of the subungual region
HP:0200097Oral mucosal blisters
Периодический (5–29%)3
HP:0000670Carious teeth
HP:0001057Aplasia cutis congenita
HP:0200041Skin erosion
Очень редкий (1–4%)1
HP:0001000Abnormality of skin pigmentation
Исключён (0%)14
HP:0000079Abnormality of the urinary system
HP:0000478Abnormality of the eye
HP:0000982Palmoplantar keratoderma
HP:0001510Growth delay
HP:0001903Anemia
HP:0001965Abnormality of the scalp
HP:0002671Basal cell carcinoma
HP:0002860Squamous cell carcinoma
HP:0004057Mitten deformity
HP:0004386Gastrointestinal inflammation
HP:0006297Enamel hypoplasia
HP:0012056Cutaneous melanoma
HP:0012252Abnormal respiratory system morphology
HP:0031464Genital blistering

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