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Late-onset junctional epidermolysis bullosa

ORPHA:79406DiseaseAutosomal recessiveAdolescent, Adult, Childhood

Ассоциированные гены (1)

COL17A1
collagen type XVII alpha 1 chain
Disease-causing germline mutation(s) in
OMIM: 113811

Фенотипы (27)

Частый (30–79%)6
HP:0001030Fragile skin
HP:0001798Anonychia
HP:0006297Enamel hypoplasia
HP:0007455Adermatoglyphia
HP:0008066Abnormal blistering of the skin
HP:0008404Nail dystrophy
Периодический (5–29%)5
HP:0000670Carious teeth
HP:0000975Hyperhidrosis
HP:0011355Localized skin lesion
HP:0020117Hypoplastic dermoepidermal hemidesmosomes
HP:0200097Oral mucosal blisters
Исключён (0%)16
HP:0000079Abnormality of the urinary system
HP:0000478Abnormality of the eye
HP:0000982Palmoplantar keratoderma
HP:0001056Milia
HP:0001075Atrophic scars
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:0010562Keloids
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