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Autosomal dominant spastic paraplegia type 9A

ORPHA:447753DiseaseAutosomal dominantAdolescent, Adult, Infancy

Ассоциированные гены (1)

ALDH18A1
aldehyde dehydrogenase 18 family member A1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 138250

Фенотипы (34)

Очень частый (80–99%)3
HP:0002064Spastic gait
HP:0002395Lower limb hyperreflexia
HP:0007350Hyperreflexia in upper limbs
Частый (30–79%)5
HP:0001761Pes cavus
HP:0003487Babinski sign
HP:0006895Lower limb hypertonia
HP:0007256Abnormal pyramidal sign
HP:0010832Abnormality of pain sensation
Периодический (5–29%)26
HP:0100515Pollakisuria
HP:0000012Urinary urgency
HP:0000020Urinary incontinence
HP:0000407Sensorineural hearing impairment
HP:0000519Developmental cataract
HP:0000666Horizontal nystagmus
HP:0000709Psychosis
HP:0000726Dementia
HP:0001250Seizure
HP:0001317Abnormal cerebellum morphology
HP:0001324Muscle weakness
HP:0001337Tremor
HP:0001653Mitral regurgitation
HP:0002166Impaired vibration sensation in the lower limbs
HP:0002172Postural instability
HP:0002280Enlarged cisterna magna
HP:0002354Memory impairment
HP:0002425Anarthria
HP:0002464Spastic dysarthria
HP:0002500Abnormal cerebral white matter morphology
HP:0002527Falls
HP:0003394Muscle spasm
HP:0003419Low back pain
HP:0007371Corpus callosum atrophy
HP:0011397Abnormality of the dorsal column of the spinal cord
HP:0012514Lower limb pain

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