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Bothnia retinal dystrophy

ORPHA:85128DiseaseAutosomal recessiveChildhood

Ассоциированные гены (1)

RLBP1
retinaldehyde binding protein 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 180090

Фенотипы (23)

Очень частый (80–99%)6
HP:0000529Progressive visual loss
HP:0000551Color vision defect
HP:0001123Visual field defect
HP:0030469Abnormal dark-adapted electroretinogram
HP:0030474Undetectable dark-adapted electroretinogram
HP:0030618Increased OCT-measured foveal thickness
Частый (30–79%)7
HP:0000493Abnormal foveal morphology
HP:0000539Abnormality of refraction
HP:0000546Retinal degeneration
HP:0000580Pigmentary retinopathy
HP:0000608Macular degeneration
HP:0007814Retinal pigment epithelial mottling
HP:0032118Retinitis
Периодический (5–29%)9
HP:0000510Rod-cone dystrophy
HP:0000603Central scotoma
HP:0000610Abnormal choroid morphology
HP:0000662Nyctalopia
HP:0001129Large central visual field defect
HP:0007722Retinal pigment epithelial atrophy
HP:0007984Electronegative electroretinogram
HP:0030528Paracentral scotoma
HP:0030529Ring scotoma
Исключён (0%)1
HP:0007737Bone spicule pigmentation of the retina

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