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Usher syndrome type 2

ORPHA:231178Clinical subtypeAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (5)

USH2A
usherin
Disease-causing germline mutation(s) in
OMIM: 608400
ADGRV1
adhesion G protein-coupled receptor V1
Disease-causing germline mutation(s) in
OMIM: 602851
MYO7A
myosin VIIA
Disease-causing germline mutation(s) in
OMIM: 276903
WHRN
whirlin
Disease-causing germline mutation(s) in
OMIM: 607928
PDZD7
PDZ domain containing 7
Modifying germline mutation in
OMIM: 612971

Фенотипы (21)

Очень частый (80–99%)7
HP:0000359Abnormality of the inner ear
HP:0000407Sensorineural hearing impairment
HP:0000510Rod-cone dystrophy
HP:0000512Abnormal electroretinogram
HP:0000572Visual loss
HP:0000575Scotoma
HP:0007730Iris hypopigmentation
Частый (30–79%)8
HP:0000518Cataract
HP:0000545Myopia
HP:0000662Nyctalopia
HP:0001133Constriction of peripheral visual field
HP:0002360Sleep abnormality
HP:0007663Reduced visual acuity
HP:0007994Peripheral visual field loss
HP:0012378Fatigue
Периодический (5–29%)5
HP:0000551Color vision defect
HP:0000716Depression
HP:0000739Anxiety
HP:0002141Gait imbalance
HP:0032036Reduced contrast sensitivity
Очень редкий (1–4%)1
HP:0001751Abnormal vestibular function

Эпидемиология (2)

Point prevalence
1-9 / 100 000
Denmark
Point prevalence
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы