04Nov 2023
Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects With Leber's Hereditary Optic Neuropathy

Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects With Leber's Hereditary Optic Neuropathy

This Phase 2 clinical trial assessed the safety, tolerability, and potential efficacy of topical elamipretide in 12 patients with Leber's Hereditary Optic Neuropathy (LHON). Administered over 52 weeks, elamipretide demonstrated overall tolerability, with mild to moderate treatment-emergent adverse events. While the primary outcome of best-corrected visual acuity (BCVA) did not show significant differences, post-hoc analysis revealed encouraging improvements in central visual field mean deviation and other visual functions during the open-label extension.

  • #ophthalmology

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