The protocol at a glance
The US label dose for people weighing at least 30 kg with Barth syndrome is 40 mg injected under the skin once a day.
| Who | Daily amount on the US label |
|---|---|
| Barth syndrome, 30 kg or more | 40 mg under the skin once a day |
| Adults with severe kidney problems (eGFR under 30, not on dialysis) | 20 mg once a day |
| Other uses | No label dose |
From the Forzinity US prescribing information (openFDA, label effective Dec 2025). Approval is an accelerated approval based on knee muscle strength, so a confirmatory trial is required.
Community protocols often use 1 to 2 mg a day for anti-aging. This is community talk, not a label dose.
What people say they do, collected from community discussions. Not tested in a trial; shown so you can compare it with the evidence above.
Mixing
No mixing table here: this one is used as a capsule, nasal spray or skin product, or has no human amount to calculate from. The general mixing calculator works for any vial.
What the trials found
Trial for the approval: 12 males with Barth syndrome, aged 12 to 35, 12 weeks of drug versus 12 weeks of placebo (crossover). Strength is the median change in knee muscle force:
Forzinity US label, Clinical Studies section. The drug was not better than placebo on the two main goals. The strength gains came in the open-label extension without a placebo group.
Side effects
- Injection-site reactions were most common in the placebo-controlled study: redness in all 12 people on the drug, pain in 75%, hardening in 67%.
- Serious allergic reactions have been reported. The vial contains benzyl alcohol, which is not for newborns.
- Blood eosinophil counts often rose, then returned to normal after 6 to 12 months.
How it works
Cardiolipin binder
The label calls it a mitochondrial cardiolipin binder. Cardiolipin is a fat found in mitochondria, the cell's power plants.
Sources
- Forzinity (elamipretide) US prescribing information via openFDA, effective Dec 2025