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ATXN1

—Ataxin 1

Chromosome: 6

Location: 6:16299112-16761491

OMIM: 601556

Ensembl: ENSG00000124788

Official·OMIM:601556 Official·ENSG00000124788

Encodes ataxin-1 protein. CAG repeat expansion causes SCA1.

Treatment Roadmap — ATXN1

Preclinical

Phase 1

Phase 2

Phase 3

Approved

Clinical trials have approximately 10% success rate. This roadmap shows the current status, not guaranteed outcomes.

Associated Diseases

Spinocerebellar Ataxia Type 1

Autosomal DominantPrevalence: 1/100000neurological

Official·OMIM:164400 Official·ORPHA98755

Summary

Clinical Trial

Approved Therapy

Registered Variant