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Fatal familial insomnia

ORPHA:466DiseaseAutosomal dominantAdult

Ассоциированные гены (1)

PRNP
prion protein (Kanno blood group)
Disease-causing germline mutation(s) in
OMIM: 176640

Фенотипы (34)

Очень частый (80–99%)4
HP:0000726Dementia
HP:0001336Myoclonus
HP:0012332Abnormal autonomic nervous system physiology
HP:0100785Insomnia
Частый (30–79%)21
HP:0000712Emotional lability
HP:0000716Depression
HP:0000738Hallucinations
HP:0000739Anxiety
HP:0000822Hypertension
HP:0000975Hyperhidrosis
HP:0001251Ataxia
HP:0001260Dysarthria
HP:0001649Tachycardia
HP:0002015Dysphagia
HP:0002071Abnormality of extrapyramidal motor function
HP:0006511Laryngeal stridor
HP:0006979Sleep-wake cycle disturbance
HP:0009926Epiphora
HP:0010535Sleep apnea
HP:0012660Thalamic hypometabolism in FDG PET
HP:0031843Bradyphrenia
HP:0033687Short term memory impairment
HP:0100543Cognitive impairment
HP:5200044Reduced attention regulation
HP:5200360Short rem sleep
Периодический (5–29%)8
HP:0000651Diplopia
HP:0001824Weight loss
HP:0001945Fever
HP:0002019Constipation
HP:0002371Loss of speech
HP:0003118Increased circulating cortisol level
HP:0007256Abnormal pyramidal sign
HP:0012689Abnormal pineal melatonin secretion
Исключён (0%)1
HP:0010850EEG with spike-wave complexes

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