Spinocerebellar ataxia type 1, or SCA1, is a rare autosomal dominant disorder with neurological symptoms including slurred speech, abnormal eye movements and ataxia of gait and stance. The cerebellar dysfunction is progressive and permanent. Symptoms normally begin between the ages of 30 and 40, though juvenile onset occurs, and death typically follows within 10 to 30 years of onset. It is caused by an expanded run of CAG repeats in the ATXN1 gene, which makes an abnormally long glutamine tract in the ataxin 1 protein and damages neurons such as Purkinje cells. No cure is known, so care focuses on managing symptoms. The source gives a worldwide estimate of 1 to 2 people per 100,000, and calls SCA1 the first spinocerebellar ataxia gene to be localized and identified.
Facts
Classification
ICD-10 ChapterVI: Diseases of the Nervous System 2 Communicable or Non-communicable Transmission Route Causative Agent
Causative Agent (category) Prevalence Band Sources
1. Spinocerebellar ataxia type 1 (Wikipedia)
Lead section
caused by an expanded number of trinucleotide repeats in the polyglutamine tract of the ATXN1 gene
Lead section [body-system]
characterized by neurological symptoms
Lead section [disease-category]
rare autosomal dominant disorder
View the Source2. Orphadata (Orphanet): Orphadata cross-references for ORPHA:98755 Spinocerebellar ataxia type 1
Orphadata (Orphanet)ICD-10 cross-referenceQuote, ICD-10 cross-reference
"Reference": "G11.8", "Source": "ICD-10"
View the Source 3. MedlinePlus Genetics (U.S. National Library of Medicine): Spinocerebellar ataxia type 1 - MedlinePlus Genetics
MedlinePlus Genetics (U.S. National Library of Medicine)Description and InheritanceQuote, Description and Inheritance
Mutations in the ATXN1 gene cause SCA1. The ATXN1 gene provides instructions for making a protein called ataxin-1. This protein is found throughout the body, but its function is unknown.
View the Source 4. Orphadata (Orphanet): Orphadata epidemiology for ORPHA:98755 Spinocerebellar ataxia type 1
Orphadata (Orphanet)Prevalence, WorldwideQuote, Prevalence, Worldwide
"PrevalenceClass": "1-9 / 100 000", "PrevalenceGeographic": "Worldwide"
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