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; PMID 33174212
- Updated
- How we grade
- 100 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 | 12 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 | 12 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 | 11 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 | 17 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
100 studies from PubMed
- Contemporary insights into elamipretide's mitochondrial mechanism of action and therapeutic effects
- Reprogramming of Treg cell-derived small extracellular vesicles effectively prevents intestinal inflammation from PANoptosis by blocking mitochondrial oxidative stress.
- Mitochondrial Cardiolipin-Targeted Tetrapeptide, SS-31, Exerts Neuroprotective Effects Within In Vitro and In Vivo Models of Spinal Cord Injury.
- Aging, mitochondrial dysfunction, and cerebral microhemorrhages: a preclinical evaluation of SS-31 (elamipretide) and development of a high-throughput machine learning-driven imaging pipeline for cerebromicrovascular protection therapeutic screening.
- The Mitochondria-Targeted Peptide Therapeutic Elamipretide Improves Cardiac and Skeletal Muscle Function During Aging Without Detectable Changes in Tissue Epigenetic or Transcriptomic Age.
- Targeting the Electron Transport System for Enhanced Longevity.
- SS-31: A promising therapeutic agent against bleomycin-induced pulmonary fibrosis in Mice.
- Beyond the injection: delivery systems reshaping retinal disease management.
- Elamipretide enhances post-thaw rooster sperm quality by mitigating oxidative stress and optimizing mitochondrial function during cryopreservation.
- Environmental enrichment highlights mitochondrial inner membrane function as a therapeutic target for sepsis-associated encephalopathy.
- Enhanced Parkin-mediated mitophagy mitigates adverse left ventricular remodelling after myocardial infarction: role of PR-364.
- ReCLAIM-2: A Randomized Phase II Clinical Trial Evaluating Elamipretide in Age-related Macular Degeneration, Geographic Atrophy Growth, Visual Function, and Ellipsoid Zone Preservation.
- Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential.
- The contribution of baseline circulating endocannabinoids to individual differences in human pain sensitivity: a quantitative sensory testing study.
- Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
- Platelet membrane-coated bio-nanoparticles of indocyanine green/elamipretide for NIR diagnosis and antioxidant therapy in acute kidney injury.
- Megalin-targeting and ROS-responsive elamipretide-conjugated polymeric prodrug for treatment of acute kidney injury.
- Comprehensive dry eye therapy: overcoming ocular surface barrier and combating inflammation, oxidation, and mitochondrial damage.
- Expanded-access use of elamipretide in a patient with membrane protein-associated neurodegeneration.
- Mitochondrial antioxidant elamipretide improves learning and memory impairment induced by chronic sleep deprivation in mice.
- Podocyte senescence: from molecular mechanisms to therapeutics.
- Mitochondria-targeted reactive oxygen species blockor SS-31 blocks hepatic stellate cell activation and alleviates hepatic fibrosis by regulating NLRP3 inflammasomes.
- Post-sepsis chronic muscle weakness can be prevented by pharmacological protection of mitochondria.
- Elamipretide(SS-31) Attenuates Idiopathic Pulmonary Fibrosis by Inhibiting the Nrf2-Dependent NLRP3 Inflammasome in Macrophages.
- Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.
- Elamipretide for Barth syndrome cardiomyopathy: gradual rebuilding of a failed power grid
- Targeting mitochondrial dysfunction with elamipretide
- Elamipretide treatment during pregnancy ameliorates the progression of polycystic kidney disease in maternal and neonatal mice with PKD1 mutations.
- Effects of Elamipretide on Autophagy in Renal Cells of Pigs with Metabolic Syndrome.
- Elamipretide for Barth syndrome cardiomyopathy: gradual rebuilding of a failed power grid.
- Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy: ReCLAIM NCGA Study.
- Long-term treatment with Elamipretide enhances healthy aging phenotypes in mice.
- A ROS-Responsive Liposomal Composite Hydrogel Integrating Improved Mitochondrial Function and Pro-Angiogenesis for Efficient Treatment of Myocardial Infarction.
- Phase 1 Clinical Trial of Elamipretide in Intermediate Age-Related Macular Degeneration and High-Risk Drusen: ReCLAIM High-Risk Drusen Study.
- N-oleoylethanolamide treatment of lymphoblasts deficient in Tafazzin improves cell growth and mitochondrial morphology and dynamics.
- 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
- The effects of a mitochondrial targeted peptide (elamipretide/SS31) on BAX recruitment and activation during apoptosis.
- SS-31 Protects Liver from Ischemia-Reperfusion Injury via Modulating Macrophage Polarization.
- Barth syndrome cardiomyopathy: targeting the mitochondria with elamipretide.
- A 50 kdyne contusion spinal cord injury with or without the drug SS-31 was not associated with major changes in muscle mass or gene expression 14 d after injury in young male mice.
- Elamipretide (SS-31) Improves Functional Connectivity in Hippocampus and Other Related Regions Following Prolonged Neuroinflammation Induced by Lipopolysaccharide in Aged Rats.
- Mitochondrial-Targeting Antioxidant SS-31 Suppresses Airway Inflammation and Oxidative Stress Induced by Cigarette Smoke.
- Mitochondrial dysfunction and beneficial effects of mitochondria-targeted small peptide SS-31 in Diabetes Mellitus and Alzheimer's disease.
- Neuroprotective Effects of a Small Mitochondrially-Targeted Tetrapeptide Elamipretide in Neurodegeneration.
- SS-31 ameliorates hepatic injury in rats subjected to severe burns plus delayed resuscitation via inhibiting the mtDNA/STING pathway in Kupffer cells.
- Antioxidant effect of Elamipretide on bull's sperm cells during freezing/thawing process.
- Role of AQP3 in the Vascular Leakage of Sepsis and the Protective Effect of Ss-31.
- 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.
- Pharmacological Blockade of PPARα Exacerbates Inflammatory Pain-Related Impairment of Spatial Memory in Rats.
- N-acylethanolamine regulation of TLR3-induced hyperthermia and neuroinflammatory gene expression: A role for PPARα.
- Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial
- Late-life restoration of mitochondrial function reverses cardiac dysfunction in old mice.
- The cardiolipin-binding peptide elamipretide mitigates fragmentation of cristae networks following cardiac ischemia reperfusion in rats.
- Restoring mitochondrial superoxide levels with elamipretide (MTP-131) protects db/db mice against progression of diabetic kidney disease.
- Mitochondria targeted peptide SS-31 prevent on cisplatin-induced acute kidney injury via regulating mitochondrial ROS-NLRP3 pathway.
- A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy.
- Elamipretide Attenuates Pyroptosis and Perioperative Neurocognitive Disorders in Aged Mice.
- The mitochondria-targeted peptide SS-31 binds lipid bilayers and modulates surface electrostatics as a key component of its mechanism of action.
- Elamipretide as a potential candidate for relieving cryodamage to human spermatozoa during cryopreservation.
- Oea Signaling Pathways and the Metabolic Benefits of Vertical Sleeve Gastrectomy.
- The mitochondria-targeting peptide elamipretide diminishes circulating HtrA2 in ST-segment elevation myocardial infarction
- Elamipretide Improves Mitochondrial Function in the Failing Human Heart
- Increased Survival Time With SS-31 After Prolonged Cardiac Arrest in Rats.
- Mitochondria-targeted cyclosporin A delivery system to treat myocardial ischemia reperfusion injury of rats.
- Effects of elamipretide on skeletal muscle in dogs with experimentally induced heart failure.
- Enhanced efficiency of mitochondria-targeted peptide SS-31 for acute kidney injury by pH-responsive and AKI-kidney targeted nanopolyplexes.
- SS-31 reduces inflammation and oxidative stress through the inhibition of Fis1 expression in lipopolysaccharide-stimulated microglia.
- Mitochondria-targeted antioxidant peptide SS-31 mediates neuroprotection in a rat experimental glaucoma model.
- Hypertension Exacerbates Cerebrovascular Oxidative Stress Induced by Mild Traumatic Brain Injury: Protective Effects of the Mitochondria-Targeted Antioxidative Peptide SS-31.
- Elamipretide (SS-31) improves mitochondrial dysfunction, synaptic and memory impairment induced by lipopolysaccharide in mice.
- Contradictory effects of mitochondria- and non-mitochondria-targeted antioxidants on hepatocarcinogenesis by altering DNA repair in mice.
- The mitochondrial antioxidant SS-31 increases SIRT1 levels and ameliorates inflammation, oxidative stress and leukocyte-endothelium interactions in type 2 diabetes.
- Protective effects of mitochondrion-targeted peptide SS-31 against hind limb ischemia-reperfusion injury.
- Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy.
- SS-31 Provides Neuroprotection by Reversing Mitochondrial Dysfunction after Traumatic Brain Injury.
- Enhancing Mitochondrial Health to Treat Hypertension.
- N-oleoylethanolamide suppresses intimal hyperplasia after balloon injury in rats through AMPK/PPARα pathway.
- Profiling plasma N-Acylethanolamine levels and their ratios as a biomarker of obesity and dysmetabolism.
- Role of mitochondrial dysfunction in renal fibrosis promoted by hypochlorite-modified albumin in a remnant kidney model and protective effects of antioxidant peptide SS-31.
- Mitochondria Protection after Acute Ischemia Prevents Prolonged Upregulation of IL-1β and IL-18 and Arrests CKD.
- Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of Elamipretide.
- Mitochondria Targeted Peptide Attenuates Mitochondrial Dysfunction, Controls Inflammation and Protects Against Spinal Cord Injury-Induced Lung Injury.
- Protective Effect of Bendavia (SS-31) Against Oxygen/Glucose-Deprivation Stress-Induced Mitochondrial Damage in Human Brain Microvascular Endothelial Cells.
- Elamipretide (SS-31) Ameliorates Isoflurane-Induced Long-Term Impairments of Mitochondrial Morphogenesis and Cognition in Developing Rats.
- SS-31 peptide enables mitochondrial targeting drug delivery: a promising therapeutic alteration to prevent hair cell damage from aminoglycosides.
- Pharmacological inhibition of FAAH modulates TLR-induced neuroinflammation, but not sickness behaviour: An effect partially mediated by central TRPV1.
- Effect of elamipretide on left ventricular function in patients with heart failure with reduced ejection fraction: the EMBRACE STEMI randomized clinical trial
- Protective Effects of Antioxidant Peptide SS-31 Against Multiple Organ Dysfunctions During Endotoxemia.
- Mitochondria-targeted peptide SS-31 attenuates renal injury via an antioxidant effect in diabetic nephropathy.
- Administration of antioxidant peptide SS-31 attenuates transverse aortic constriction-induced pulmonary arterial hypertension in mice.
- BDNF pathway is involved in the protective effects of SS-31 on isoflurane-induced cognitive deficits in aging mice.
- Targeting CD36-mediated inflammation reduces acute brain injury in transient, but not permanent, ischemic stroke.
- SS-31 attenuates TNF-α induced cytokine release from C2C12 myotubes.
- A mitochondrial therapeutic reverses visual decline in mouse models of diabetes.
- Mitochondrion-Targeted Peptide SS-31 Inhibited Oxidized Low-Density Lipoproteins-Induced Foam Cell Formation through both ROS Scavenging and Inhibition of Cholesterol Influx in RAW264.7 Cells.
- Gastric bypass in morbid obese patients is associated with reduction in adipose tissue inflammation via N-oleoylethanolamide (OEA)-mediated pathways.
- Synthesis and evaluation of fatty acid amides on the N-oleoylethanolamide-like activation of peroxisome proliferator activated receptor α.
- Mitochondria-targeted antioxidant promotes recovery of skeletal muscle mitochondrial function after burn trauma assessed by in vivo 31P nuclear magnetic resonance and electron paramagnetic resonance spectroscopy.
- Mitochondrial targeted antioxidant Peptide ameliorates hypertensive cardiomyopathy.
- Novel mitochondria-targeted antioxidant peptide ameliorates burn-induced apoptosis and endoplasmic reticulum stress in the skeletal muscle of mice.