Side by side

1. 1.Binds cardiolipin, the lipid that anchors the energy-producing machinery of the inner mitochondrial membrane.
2. 2.Stabilises the electron transport chain, improving ATP output in damaged mitochondria.
3. 3.Reduces reactive oxygen species leaking from the chain.
4. 4.Restores mitochondrial inner-membrane architecture in laboratory models.
5. 5.Because it acts on the organelle rather than a receptor, its effects are broad rather than tissue-specific — both the appeal and the problem.

• Barth syndrome, a rare genetic mitochondrial disorder
• Primary mitochondrial myopathy
• Heart failure with reduced ejection fraction
• Dry age-related macular degeneration
• Ischaemia-reperfusion injury

• Improved walking distance in some mitochondrial myopathy trials, though the largest trial missed its primary endpoint
• Improved cardiac measures in small heart-failure studies
• Lower oxidative-stress markers
• No consistent, confirmed clinical benefit yet

• Injection-site reactions
• Headache, nausea, dizziness
• Transient blood-pressure changes during infusion
• Long-term safety unknown

Research context only — protocols exist only inside trials: daily subcutaneous 40 mg in Barth syndrome studies, and intravenous infusions, commonly around 0.25 mg per kilogram per hour, in cardiac and eye trials. There is no validated self-administered regimen, and trial doses bear no relation to the syringe units quoted on vendor charts.

Research context only