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Hemihyperplasia-multiple lipomatosis syndrome

ORPHA:276280Malformation syndromeNot applicableChildhood

Ассоциированные гены (1)

PIK3CA
phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha
Disease-causing somatic mutation(s) in
OMIM: 171834

Фенотипы (19)

Облигатный (100%)1
HP:0001548Overgrowth
Частый (30–79%)2
HP:0004099Macrodactyly
HP:0100659Abnormality of the cerebral vasculature
Периодический (5–29%)15
HP:0000034Hydrocele testis
HP:0000105Enlarged kidney
HP:0001012Multiple lipomas
HP:0001051Seborrheic dermatitis
HP:0001829Foot polydactyly
HP:0002624Abnormal venous morphology
HP:0002650Scoliosis
HP:0003764Nevus
HP:0008551Microtia
HP:00107142-4 toe syndactyly
HP:0012887Ovarian serous cystadenoma
HP:0040009Hyperparakeratosis
HP:0100578Lipoatrophy
HP:0100585Telangiectasia of the skin
HP:0100763Abnormality of the lymphatic system
Очень редкий (1–4%)1
HP:0002667Nephroblastoma

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