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Kindler epidermolysis bullosa

ORPHA:2908DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

FERMT1
FERM domain containing kindlin 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 607900

Фенотипы (41)

Очень частый (80–99%)7
HP:0000982Palmoplantar keratoderma
HP:0000992Cutaneous photosensitivity
HP:0001029Poikiloderma
HP:0008065Aplasia/Hypoplasia of the skin
HP:0008066Abnormal blistering of the skin
HP:0010783Erythema
HP:0100825Cheilitis
Частый (30–79%)15
HP:0000230Gingivitis
HP:0000670Carious teeth
HP:0000682Abnormality of dental enamel
HP:0000704Periodontitis
HP:0001000Abnormality of skin pigmentation
HP:0001581Recurrent skin infections
HP:0001741Phimosis
HP:0002015Dysphagia
HP:0002043Esophageal stricture
HP:0002583Colitis
HP:0006101Finger syndactyly
HP:0006323Premature loss of primary teeth
HP:0008388Abnormal toenail morphology
HP:0100490Camptodactyly of finger
HP:0100633Esophagitis
Периодический (5–29%)19
HP:0000262Turricephaly
HP:0000509Conjunctivitis
HP:0000656Ectropion
HP:0000772Abnormal rib morphology
HP:0000929Abnormal skull morphology
HP:0000962Hyperkeratosis
HP:0000987Atypical scarring of skin
HP:0001056Milia
HP:0001371Flexion contracture
HP:0001602Laryngeal stenosis
HP:0001903Anemia
HP:0002037Inflammation of the large intestine
HP:0002860Squamous cell carcinoma
HP:0004378Abnormality of the anus
HP:0007957Corneal opacity
HP:0010044Short 4th metacarpal
HP:0010047Short 5th metacarpal
HP:0012227Urethral stricture
HP:0100517Neoplasm of the urethra

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
1-9 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы