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Muckle-Wells syndrome

ORPHA:575DiseaseAutosomal dominantChildhood, Infancy, Neonatal

Ассоциированные гены (1)

NLRP3
NLR family pyrin domain containing 3
Disease-causing germline mutation(s) in
OMIM: 606416

Фенотипы (37)

Очень частый (80–99%)11
HP:0000408Progressive sensorineural hearing impairment
HP:0000509Conjunctivitis
HP:0000554Uveitis
HP:0000988Skin rash
HP:0001369Arthritis
HP:0001744Splenomegaly
HP:0001769Broad foot
HP:0002240Hepatomegaly
HP:0002829Arthralgia
HP:0006824Cranial nerve paralysis
HP:0100534Episcleritis
Частый (30–79%)7
HP:0000100Nephrotic syndrome
HP:0000112Nephropathy
HP:0001025Urticaria
HP:0001917Renal amyloidosis
HP:0001939Abnormality of metabolism/homeostasis
HP:0002027Abdominal pain
HP:0003565Elevated erythrocyte sedimentation rate
Периодический (5–29%)19
HP:0000078Abnormality of the genital system
HP:0000174Abnormal palate morphology
HP:0000256Macrocephaly
HP:0000366Abnormality of the nose
HP:0000501Glaucoma
HP:0000648Optic atrophy
HP:0000823Delayed puberty
HP:0001608Abnormality of the voice
HP:0001761Pes cavus
HP:0001903Anemia
HP:0001945Fever
HP:0002091Restrictive ventilatory defect
HP:0002633Vasculitis
HP:0003326Myalgia
HP:0004299Hernia of the abdominal wall
HP:0004322Short stature
HP:0008064Ichthyosis
HP:0011107Recurrent aphthous stomatitis
HP:0100490Camptodactyly of finger

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы