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Autosomal dominant spastic paraplegia type 17

ORPHA:100998DiseaseAutosomal dominantAdolescent, Adult, Childhood

Ассоциированные гены (1)

BSCL2
BSCL2 lipid droplet biogenesis associated, seipin
Disease-causing germline mutation(s) in
OMIM: 606158

Фенотипы (16)

Частый (30–79%)6
HP:0001347Hyperreflexia
HP:0001436Abnormality of the foot musculature
HP:0002064Spastic gait
HP:0003487Babinski sign
HP:0009027Foot dorsiflexor weakness
HP:0009130Hand muscle atrophy
Периодический (5–29%)10
HP:0001171Split hand
HP:0001763Pes planus
HP:0002174Postural tremor
HP:0002936Distal sensory impairment
HP:0003693Distal amyotrophy
HP:0030237Hand muscle weakness
HP:0030838Hip pain
HP:0030839Knee pain
HP:0031374Ankle weakness
HP:0040131Abnormal motor nerve conduction velocity

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