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Autosomal spastic paraplegia type 18

ORPHA:209951DiseaseAutosomal dominant, Autosomal recessiveChildhood, Infancy

Ассоциированные гены (1)

ERLIN2
ER lipid raft associated 2
Disease-causing germline mutation(s) in
OMIM: 611605

Фенотипы (34)

Очень частый (80–99%)11
HP:0001249Intellectual disability
HP:0001263Global developmental delay
HP:0001347Hyperreflexia
HP:0001371Flexion contracture
HP:0002460Distal muscle weakness
HP:0002540Inability to walk
HP:0002987Elbow flexion contracture
HP:0006380Knee flexion contracture
HP:0006466Ankle flexion contracture
HP:0007350Hyperreflexia in upper limbs
HP:0012453Bilateral wrist flexion contracture
Частый (30–79%)6
HP:0000154Wide mouth
HP:0000158Macroglossia
HP:0001257Spasticity
HP:0001344Absent speech
HP:0002079Hypoplasia of the corpus callosum
HP:0003487Babinski sign
Периодический (5–29%)17
HP:0000218High palate
HP:0000322Short philtrum
HP:0000377Abnormal pinna morphology
HP:0000574Thick eyebrow
HP:0000664Synophrys
HP:0000666Horizontal nystagmus
HP:0001250Seizure
HP:0001583Rotary nystagmus
HP:0002015Dysphagia
HP:0002166Impaired vibration sensation in the lower limbs
HP:0002378Hand tremor
HP:0003273Hip contracture
HP:0003306Spinal rigidity
HP:0005830Flexion contracture of toe
HP:0005997Restricted neck movement due to contractures
HP:0011448Ankle clonus
HP:0100712Abnormal lumbar spine morphology

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