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Isolated megalencephaly

ORPHA:2477Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

TBC1D7
TBC1 domain family member 7
Disease-causing germline mutation(s) (loss of function) in
OMIM: 612655

Фенотипы (16)

Очень частый (80–99%)12
HP:0000235Abnormality of the fontanelles or cranial sutures
HP:0000256Macrocephaly
HP:0000268Dolichocephaly
HP:0000269Prominent occiput
HP:0000307Pointed chin
HP:0000431Wide nasal bridge
HP:0000470Short neck
HP:0000490Deeply set eye
HP:0001249Intellectual disability
HP:0001956Truncal obesity
HP:0002007Frontal bossing
HP:0002750Delayed skeletal maturation
Частый (30–79%)4
HP:0000040Long penis
HP:0000053Macroorchidism
HP:0001631Atrial septal defect
HP:0002857Genu valgum

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы