
In a Phase 2 clinical trial, the safety, tolerability, and potential efficacy of topical elamipretide were assessed in 12 Leber’s Hereditary Optic Neuropathy (LHON) patients over 52 weeks, followed by a 108-week open extension. While elamipretide was well-tolerated, no significant improvement in best corrected visual acuity (BCVA) was observed. However, post-hoc analysis revealed encouraging improvements in central visual field mean deviation.
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