Expanded-Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series.

Plain Language Summary

Two patients with rare, progressive mitochondrial diseases affecting their eyes were treated with elamipretide under an expanded-access program. Both patients tolerated the peptide well and reported improvement in their symptoms while on therapy. This case series adds to growing evidence that elamipretide may benefit patients with mitochondrial disorders that cause vision problems.