SS-31 Research for Barth Syndrome
An evidence-based overview of research examining SS-31 in the context of barth syndrome. This page synthesizes findings from peer-reviewed literature.
Research Summary
Barth syndrome is caused by TAZ gene mutations affecting cardiolipin remodeling, creating a direct mechanistic rationale for SS-31. In the Phase 2/3 TAZPOWER trial (n=12), the crossover phase did not meet primary endpoints, but the open-label extension showed a significant ~96-meter improvement in 6-minute walk test at 36 weeks plus improvements in muscle strength and fatigue.
Referenced Studies
Click any PMID to view the full study on PubMed.
Important Disclaimer
This page summarizes research findings and does not constitute medical advice. SS-31 may have regulatory approval for some indications but should only be used under qualified medical supervision. Always consult a healthcare provider before making health decisions.