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Primary lateral sclerosis

ORPHA:35689DiseaseAutosomal dominant, Autosomal recessive, Not applicableAdult, Elderly

Ассоциированные гены (1)

SPG7
SPG7 matrix AAA peptidase subunit, paraplegin
Disease-causing germline mutation(s) in
OMIM: 602783

Фенотипы (26)

Очень частый (80–99%)5
HP:0002493Upper motor neuron dysfunction
HP:0003487Babinski sign
HP:0007034Generalized hyperreflexia
HP:0001257Spasticity
HP:0002127Abnormal upper motor neuron morphology
Частый (30–79%)13
HP:0000012Urinary urgency
HP:0002015Dysphagia
HP:0002064Spastic gait
HP:0002141Gait imbalance
HP:0002200Pseudobulbar signs
HP:0002311Incoordination
HP:0002371Loss of speech
HP:0002464Spastic dysarthria
HP:0003444EMG: chronic denervation signs
HP:0007199Progressive spastic paraparesis
HP:0007772Impaired smooth pursuit
HP:0010549Weakness due to upper motor neuron dysfunction
HP:0031993Hoffmann sign
Периодический (5–29%)5
HP:0001611Hypernasal speech
HP:0006827Atrophy of the spinal cord
HP:0007002Motor axonal neuropathy
HP:0010873Cervical spinal cord atrophy
HP:0100543Cognitive impairment
Исключён (0%)3
HP:0002071Abnormality of extrapyramidal motor function
HP:0002366Abnormal lower motor neuron morphology
HP:0003474Somatic sensory dysfunction

Эпидемиология (1)

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