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Spinocerebellar ataxia type 25

ORPHA:101111DiseaseAutosomal dominantAll ages

Ассоциированные гены (2)

SCA25
spinocerebellar ataxia 25
Disease-causing germline mutation(s) in
PNPT1
polyribonucleotide nucleotidyltransferase 1
Disease-causing germline mutation(s) in
OMIM: 610316

Фенотипы (23)

Очень частый (80–99%)1
HP:0002073Progressive cerebellar ataxia
Частый (30–79%)19
HP:0007328Impaired pain sensation
HP:0007663Reduced visual acuity
HP:0011468Facial tics
HP:0031422Abnormal morphology of the cerebellar cortex
HP:0100275Diffuse cerebellar atrophy
HP:0000012Urinary urgency
HP:0000486Strabismus
HP:0000639Nystagmus
HP:0000763Sensory neuropathy
HP:0001761Pes cavus
HP:0002066Gait ataxia
HP:0002464Spastic dysarthria
HP:0002522Areflexia of lower limbs
HP:0002650Scoliosis
HP:0002936Distal sensory impairment
HP:0003387Decreased number of large peripheral myelinated nerve fibers
HP:0003445EMG: neuropathic changes
HP:0003487Babinski sign
HP:0006937Impaired distal tactile sensation
Периодический (5–29%)3
HP:0000317Facial myokymia
HP:0002013Vomiting
HP:0002574Episodic abdominal 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