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Autosomal recessive spastic paraplegia type 21

ORPHA:101001DiseaseAutosomal recessiveAdolescent, Adult, Childhood

Ассоциированные гены (1)

SPG21
SPG21 abhydrolase domain containing, maspardin
Disease-causing germline mutation(s) in
OMIM: 608181

Фенотипы (19)

Очень частый (80–99%)3
HP:0001288Gait disturbance
HP:0000726Dementia
HP:0007256Abnormal pyramidal sign
Частый (30–79%)14
HP:0001257Spasticity
HP:0001263Global developmental delay
HP:0001268Mental deterioration
HP:0001288Gait disturbance
HP:0001347Hyperreflexia
HP:0002015Dysphagia
HP:0002079Hypoplasia of the corpus callosum
HP:0002186Apraxia
HP:0002476Primitive reflex
HP:0003134Abnormality of peripheral nerve conduction
HP:0006892Frontotemporal cerebral atrophy
HP:0007340Lower limb muscle weakness
HP:0010526Dysgraphia
HP:0012075Personality disorder
Периодический (5–29%)2
HP:0001317Abnormal cerebellum morphology
HP:0002071Abnormality of extrapyramidal motor function

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