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Lipoid proteinosis

ORPHA:530Malformation syndromeAutosomal recessiveAll ages

Ассоциированные гены (1)

ECM1
extracellular matrix protein 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602201

Фенотипы (23)

Очень частый (80–99%)12
HP:0000168Abnormality of the gingiva
HP:0000179Thick lower lip vermilion
HP:0000199Tongue nodules
HP:0001061Acne
HP:0001072Thickened skin
HP:0001482Subcutaneous nodule
HP:0001609Hoarse voice
HP:0008066Abnormal blistering of the skin
HP:0011830Abnormal oral mucosa morphology
HP:0100699Scarring
HP:0200034Papule
HP:0200039Pustule
Частый (30–79%)8
HP:0000171Microglossia
HP:0000218High palate
HP:0000962Hyperkeratosis
HP:0001332Dystonia
HP:0002015Dysphagia
HP:0002205Recurrent respiratory infections
HP:0002293Alopecia of scalp
HP:0200043Verrucae
Периодический (5–29%)3
HP:0001250Seizure
HP:0002514Cerebral calcification
HP:0100582Nasal polyposis

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