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FXN

—Frataxin

Chromosome: 9

Location: 9:69035730-69079076

OMIM: 606829

Ensembl: ENSG00000165060

Official·OMIM:606829 Official·ENSG00000165060

Encodes frataxin protein. GAA repeat expansion causes Friedreich ataxia.

Treatment Roadmap — FXN

Preclinical

Phase 1

4 trial

Phase 2

2 trial

Phase 3

1 trial

Approved

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

Associated Diseases

Friedreich Ataxia

Autosomal RecessivePrevalence: 1/50000neurological

Official·OMIM:229300 Official·ORPHA95

Summary

Clinical Trial

Approved Therapy

Registered Variant