Condition
Barth Syndrome
Barth syndrome is a rare genetic condition in which a faulty TAFAZZIN gene disrupts cardiolipin, a fat mitochondria need to make energy, leaving muscles and the heart weak. Elamipretide has the strongest evidence, and in a long-term follow-up people walked about 96 meters farther in six minutes than at the start. That follow-up was open-label with only 10 people, and oleoylethanolamide has very limited support, from lab-grown cells alone.
Sources: PMID 38602181
- Updated
- How we grade
- 13 studies cited
- Supplements studied
- 1
- Medicines and peptides
- 1
- Graded outcomes
- 5
Evidence by supplement
- Strong
- Moderate
- Limited
- Very limited
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Mitochondrial Function (Barth Syndrome) An in vitro study found OEA treatment of Tafazzin-deficient lymphoblasts (a model of Barth syndrome) improved cell growth, mitochondrial morphology, and mitochondrial dynamics (PMID:35676289). No in vivo or clinical data available. | Improves (the measure goes up) | Small effect | 1 study | |||
Studies that measured mitochondrial function (barth syndrome) | ||||||
Prescription medicines and peptides studied, for context
Elamipretide (Forzinity/SS-31)
- 6-Minute Walk Test (Barth Syndrome): improves
- Muscle Strength (Barth Syndrome): improves
- Safety and Tolerability: improves
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| 6-Minute Walk Test (Barth Syndrome) TAZPOWER 168-week OLE (n=10): Cumulative 96.1 meter improvement in 6MWT from baseline (P=0.003). Significant improvements maintained at all OLE timepoints. Led to FDA accelerated approval in 2025. | Improves (the measure goes up) | Large effect | 8 studies | |||
| Muscle Strength (Barth Syndrome) TAZPOWER trial: Knee extensor muscle strength improved by >45% with elamipretide treatment. Significantly correlated with improvement in 6MWT. First approved therapy for Barth syndrome. | Improves (the measure goes up) | Large effect | 8 studies | |||
| Safety and Tolerability TAZPOWER 168-week OLE: Well tolerated with injection-site reactions as most common adverse event. No serious safety signals. FDA granted Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Disease designations. | Improves (the measure goes up) | Large effect | 7 studies | |||
| Cardiac Function (Barth Syndrome) TAZPOWER trial demonstrated improvements in cardiac function in Barth syndrome cardiomyopathy. Elamipretide stabilizes mitochondrial cristae and improves bioenergetics in cardiomyocytes. Long-term safety data favorable over 168 weeks. | Improves (the measure goes up) | Moderate effect | 12 studies |
Key findings
- Mitochondrial Function (Barth Syndrome)Improves (the measure goes up)
Safety notes in the studies
- TAZPOWER 168-week OLE: Well tolerated with injection-site reactions as most common adverse event.
Studies cited
13 studies from PubMed
- Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential.
- ReCLAIM-2: A Randomized Phase II Clinical Trial Evaluating Elamipretide in Age-related Macular Degeneration, Geographic Atrophy Growth, Visual Function, and Ellipsoid Zone Preservation.
- Contemporary insights into elamipretide's mitochondrial mechanism of action and therapeutic effects
- Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
- N-oleoylethanolamide treatment of lymphoblasts deficient in Tafazzin improves cell growth and mitochondrial morphology and dynamics.
- Phase 1 Clinical Trial of Elamipretide in Intermediate Age-Related Macular Degeneration and High-Risk Drusen: ReCLAIM High-Risk Drusen Study.
- A ROS-Responsive Liposomal Composite Hydrogel Integrating Improved Mitochondrial Function and Pro-Angiogenesis for Efficient Treatment of Myocardial Infarction.
- A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.
- Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial
- The mitochondria-targeting peptide elamipretide diminishes circulating HtrA2 in ST-segment elevation myocardial infarction
- A randomized, placebo-controlled clinical trial evaluating of a mouthwash containing Sambucus williamsii var. coreana extract for prevention of gingivitits.
- Evaluating the efficacy of curcumin in the management of oral potentially malignant disorders: a systematic review and meta-analysis.
- Effects of Aloe Vera in the Treatment of Oral Ulcers: A Systematic Review and Meta-Analysis of Randomised Controlled Trials.