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Maffucci syndrome

ORPHA:163634DiseaseNot applicableChildhood

Ассоциированные гены (2)

IDH2
isocitrate dehydrogenase (NADP(+)) 2
Disease-causing somatic mutation(s) in
OMIM: 147650
IDH1
isocitrate dehydrogenase (NADP(+)) 1
Disease-causing somatic mutation(s) in
OMIM: 147700

Фенотипы (24)

Очень частый (80–99%)4
HP:0002797Osteolysis
HP:0004936Venous thrombosis
HP:0005701Multiple enchondromatosis
HP:0007461Hemangiomatosis
Частый (30–79%)5
HP:0001482Subcutaneous nodule
HP:0002650Scoliosis
HP:0002653Bone pain
HP:0004322Short stature
HP:0100777Exostoses
Периодический (5–29%)15
HP:0000853Goiter
HP:0001510Growth delay
HP:0002015Dysphagia
HP:0002757Recurrent fractures
HP:0002893Pituitary adenoma
HP:0002897Parathyroid adenoma
HP:0003002Breast carcinoma
HP:0006765Chondrosarcoma
HP:0006824Cranial nerve paralysis
HP:0009592Astrocytoma
HP:0100021Cerebral palsy
HP:0100242Sarcoma
HP:0100615Ovarian neoplasm
HP:0100641Neoplasm of the adrenal cortex
HP:0100733Neoplasm of the parathyroid gland

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