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Autosomal recessive spastic paraplegia type 67

ORPHA:401820DiseaseAutosomal recessiveChildhood, Infancy

Ассоциированные гены (1)

PGAP1
post-GPI attachment to proteins inositol deacylase 1
Disease-causing germline mutation(s) in
OMIM: 611655

Фенотипы (15)

Частый (30–79%)15
HP:0001256Intellectual disability, mild
HP:0001263Global developmental delay
HP:0001274Agenesis of corpus callosum
HP:0001288Gait disturbance
HP:0001347Hyperreflexia
HP:0002061Lower limb spasticity
HP:0002064Spastic gait
HP:0002120Cerebral cortical atrophy
HP:0003487Babinski sign
HP:0003700Generalized amyotrophy
HP:0006817Aplasia/Hypoplasia of the cerebellar vermis
HP:0007020Progressive spastic paraplegia
HP:0012447Abnormal myelination
HP:0100022Abnormality of movement
HP:0200085Limb tremor

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