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Localized dystrophic epidermolysis bullosa, acral form

ORPHA:158673Clinical subtypeAutosomal 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 (20)

Частый (30–79%)17
HP:0000963Thin skin
HP:0000972Palmoplantar hyperkeratosis
HP:0000992Cutaneous photosensitivity
HP:0001009Telangiectasia
HP:0001029Poikiloderma
HP:0001030Fragile skin
HP:0001056Milia
HP:0001810Dystrophic toenail
HP:0002043Esophageal stricture
HP:0002814Abnormality of the lower limb
HP:0004334Dermal atrophy
HP:0007473Crusting erythematous dermatitis
HP:0008066Abnormal blistering of the skin
HP:0008391Dystrophic fingernails
HP:0010783Erythema
HP:0025474Erythematous plaque
HP:0031045Acral blistering
Периодический (5–29%)3
HP:0002815Abnormality of the knee
HP:0008390Recurrent loss of toenails and fingernails
HP:0009811Abnormality of the elbow

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