We recently completed a MARA Rating for a treatment for Barth syndrome — a rare, life-threatening disease that affects young men and has no established disease-modifying therapy.

The randomized trial answered one question: is this treatment significantly better than placebo on measures of exercise capacity and fatigue? The answer was no. The primary endpoints were not met.

Regulators and access bodies face a different question: should patients with no alternatives be denied a treatment, even when the evidence is inconclusive?

These questions do not have the same answer. The trial addressed the first. The disease context shapes the second.

Read the full MARA Rating here: https://mararating.com/report/forzinity-elamipretide-for-barth-syndrome-as-of-february-2026/