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Autosomal recessive spastic paraplegia type 66

ORPHA:401815DiseaseAutosomal recessiveInfancy

Ассоциированные гены (1)

ARSI
arylsulfatase family member I
Disease-causing germline mutation(s) in
OMIM: 610009

Фенотипы (14)

Частый (30–79%)14
HP:0001288Gait disturbance
HP:0001301Chronic sensorineural polyneuropathy
HP:0001321Cerebellar hypoplasia
HP:0001762Talipes equinovarus
HP:0002061Lower limb spasticity
HP:0002064Spastic gait
HP:0002079Hypoplasia of the corpus callosum
HP:0002166Impaired vibration sensation in the lower limbs
HP:0002509Limb hypertonia
HP:0007020Progressive spastic paraplegia
HP:0007210Lower limb amyotrophy
HP:0030048Colpocephaly
HP:0001249Intellectual disability
HP:0001284Areflexia

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