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Familial drusen

ORPHA:75376DiseaseAutosomal dominantAdult

Ассоциированные гены (3)

CFI
complement factor I
Disease-causing germline mutation(s) in
OMIM: 217030
CFH
complement factor H
Disease-causing germline mutation(s) in
OMIM: 134370
EFEMP1
EGF-like fibulin extracellular matrix protein 1
Disease-causing germline mutation(s) in
OMIM: 601548

Фенотипы (20)

Облигатный (100%)2
HP:0030499Macular drusen
HP:0030500Yellow/white lesions of the macula
Очень частый (80–99%)3
HP:0000572Visual loss
HP:0007754Macular dystrophy
HP:0007937Reticular pigmentary degeneration
Частый (30–79%)6
HP:0007703Abnormality of retinal pigmentation
HP:0011509Macular hyperpigmentation
HP:0012508Metamorphopsia
HP:0030629Perifoveal ring of hyperautofluorescence
HP:0030631Hyperautofluorescent macular lesion
HP:0030632Hypoautofluorescent macular lesion
Периодический (5–29%)9
HP:0000613Photophobia
HP:0007401Macular atrophy
HP:0007793Granular macular appearance
HP:0007950Peripapillary chorioretinal atrophy
HP:0011506Choroidal neovascularization
HP:0012231Exudative retinal detachment
HP:0025574Macular hemorrhage
HP:0030528Paracentral scotoma
HP:0031526Subretinal fluid

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы