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Segmental progressive overgrowth syndrome with fibroadipose hyperplasia

ORPHA:314662DiseaseNot applicableAntenatal, Neonatal

Ассоциированные гены (1)

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

Фенотипы (14)

Облигатный (100%)1
HP:0001548Overgrowth
Частый (30–79%)6
HP:0009126Increased adipose tissue
HP:0011276Vascular skin abnormality
HP:0012032Lipoma
HP:0025104Capillary malformation
HP:0100578Lipoatrophy
HP:0100774Hyperostosis
Периодический (5–29%)7
HP:0000034Hydrocele testis
HP:0002019Constipation
HP:0010442Polydactyly
HP:0010816Epidermal nevus
HP:0012725Cutaneous syndactyly
HP:0030424Epididymal cyst
HP:0100559Lower limb asymmetry

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