About Spinocerebellar Ataxia
Spinocerebellar ataxias (SCAs) are a heterogeneous group of autosomal dominant neurodegenerative disorders characterized by progressive cerebellar ataxia, with over 40 genetic subtypes identified. The most common types (SCA1, SCA2, SCA3, SCA6, SCA7) are caused by CAG trinucleotide repeat expansions in their respective genes, encoding polyglutamine tracts that cause toxic protein aggregation. Clinical features beyond cerebellar ataxia vary by subtype and include pyramidal signs, peripheral neuropathy, ophthalmoplegia, and cognitive decline.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Spinocerebellar Ataxia trials.
SCA subtype must be genetically confirmed — repeat expansion length and subtype are required for all trials
Scale for Assessment and Rating of Ataxia (SARA) score is the primary eligibility and outcome measure
CAG repeat length predicts age of onset and progression rate — this affects trial stratification and eligibility windows
Antisense oligonucleotide (ASO) and gene silencing trials are subtype-specific (e.g., SCA3 ASO trials differ from SCA1)
Patient Resources
Natural History Registry
Clinical Research Consortium for Spinocerebellar Ataxias (CRC-SCA) Registry
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