The information on this page is for informational purposes only and does not constitute medical advice. Consult your genetic counselor or specialist physician for treatment decisions.

ATXN3

—Ataxin 3

Chromosome: 14

Location: 14:92044482-92106625

OMIM: 607047

Ensembl: ENSG00000066427

Official·OMIM:607047 Official·ENSG00000066427

Encodes ataxin-3 protein. CAG repeat expansion causes SCA3 (Machado-Joseph).

Treatment Roadmap — ATXN3

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 3 (Machado-Joseph)

Autosomal DominantPrevalence: 1/100000neurological

Official·OMIM:109150 Official·ORPHA98757

Summary

Clinical Trial

Approved Therapy

Registered Variant