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Autosomal dominant optic atrophy and cataract

ORPHA:67036DiseaseAutosomal dominantChildhood

Ассоциированные гены (1)

OPA3
outer mitochondrial membrane lipid metabolism regulator OPA3
Disease-causing germline mutation(s) in
OMIM: 606580

Фенотипы (35)

Облигатный (100%)1
HP:0000505Visual impairment
Очень частый (80–99%)2
HP:0000648Optic atrophy
HP:0007663Reduced visual acuity
Частый (30–79%)14
HP:0000518Cataract
HP:0000603Central scotoma
HP:0000639Nystagmus
HP:0001251Ataxia
HP:0001272Cerebellar atrophy
HP:0001284Areflexia
HP:0002174Postural tremor
HP:0002317Unsteady gait
HP:0002522Areflexia of lower limbs
HP:0003394Muscle spasm
HP:0003401Paresthesia
HP:0003474Somatic sensory dysfunction
HP:0010924Posterior cortical cataract
HP:0012531Pain
Периодический (5–29%)18
HP:0000552Tritanomaly
HP:0000618Blindness
HP:0000642Red-green dyschromatopsia
HP:0001172Abnormal thumb morphology
HP:0001315Reduced tendon reflexes
HP:0001377Limited elbow extension
HP:0001761Pes cavus
HP:0002322Resting tremor
HP:0002403Positive Romberg sign
HP:0003438Absent Achilles reflex
HP:0006248Limited wrist movement
HP:0007076Extrapyramidal muscular rigidity
HP:0007787Posterior subcapsular cataract
HP:0007795Anterior cortical cataract
HP:0007976Cerulean cataract
HP:0009468Deviation of the 2nd finger
HP:0010522Dyslexia
HP:0010923Anterior subcapsular cataract

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