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Alexander disease type II

ORPHA:363722Clinical subtypeAutosomal dominantAdolescent, Adult, Childhood

Ассоциированные гены (1)

GFAP
glial fibrillary acidic protein
Disease-causing germline mutation(s) (gain of function) in
OMIM: 137780

Фенотипы (20)

Очень частый (80–99%)2
HP:0010873Cervical spinal cord atrophy
HP:0011441Abnormality of the medulla oblongata
Частый (30–79%)15
HP:0000639Nystagmus
HP:0001251Ataxia
HP:0001257Spasticity
HP:0001260Dysarthria
HP:0001347Hyperreflexia
HP:0001618Dysphonia
HP:0002015Dysphagia
HP:0002313Spastic paraparesis
HP:0002518Abnormal periventricular white matter morphology
HP:0002839Urinary bladder sphincter dysfunction
HP:0003487Babinski sign
HP:0003690Limb muscle weakness
HP:0007109Periventricular cysts
HP:0010530Palatal myoclonus
HP:0012332Abnormal autonomic nervous system physiology
Периодический (5–29%)3
HP:0002063Rigidity
HP:0002360Sleep abnormality
HP:0002650Scoliosis

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