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Focal facial dermal dysplasia type IV

ORPHA:398189Clinical subtypeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

CYP26C1
cytochrome P450 family 26 subfamily C member 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 608428

Фенотипы (17)

Очень частый (80–99%)3
HP:0004426Abnormality of the cheek
HP:0008066Abnormal blistering of the skin
HP:3000019Abnormality of buccal mucosa
Частый (30–79%)14
HP:0000252Microcephaly
HP:0000331Short chin
HP:0001028Hemangioma
HP:0001269Hemiparesis
HP:0002170Intracranial hemorrhage
HP:0003764Nevus
HP:0007359Focal-onset seizure
HP:0011124Abnormality of epidermal morphology
HP:0025167Fragmented elastic fibers in the dermis
HP:0100494Abnormal mast cell morphology
HP:0100699Scarring
HP:0000175Cleft palate
HP:0000204Cleft upper lip
HP:0000238Hydrocephalus

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