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Autosomal recessive spastic paraplegia type 26

ORPHA:101006DiseaseAutosomal recessiveAdolescent, Childhood

Ассоциированные гены (1)

B4GALNT1
beta-1,4-N-acetyl-galactosaminyltransferase 1
Disease-causing germline mutation(s) in
OMIM: 601873

Фенотипы (22)

Частый (30–79%)10
HP:0001249Intellectual disability
HP:0001288Gait disturbance
HP:0001324Muscle weakness
HP:0001347Hyperreflexia
HP:0002061Lower limb spasticity
HP:0002120Cerebral cortical atrophy
HP:0003202Skeletal muscle atrophy
HP:0003487Babinski sign
HP:0007141Sensorimotor neuropathy
HP:0030890Hyperintensity of cerebral white matter on MRI
Периодический (5–29%)9
HP:0000079Abnormality of the urinary system
HP:0000518Cataract
HP:0001317Abnormal cerebellum morphology
HP:0001332Dystonia
HP:0001761Pes cavus
HP:0002650Scoliosis
HP:0006938Impaired vibration sensation at ankles
HP:0007024Pseudobulbar paralysis
HP:0100660Dyskinesia
Очень редкий (1–4%)3
HP:0001265Hyporeflexia
HP:0008209Premature ovarian insufficiency
HP:0040171Decreased serum testosterone concentration

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