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TAFAZZIN

—Tafazzin

Chromosome: X

Location: X:154409456-154421726

OMIM: 300394

Ensembl: ENSG00000102125

Official·OMIM:300394 Official·ENSG00000102125

Encodes tafazzin enzyme. Mutations cause Barth syndrome.

Treatment Roadmap — TAFAZZIN

Preclinical

Phase 1

Phase 2

2 trial

Phase 3

Approved

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

Associated Diseases

Barth Syndrome

X-Linked RecessivePrevalence: 1/300000cardiac

Official·OMIM:302060 Official·ORPHA111

Summary

Clinical Trial

Approved Therapy

Registered Variant