Can a Mitochondria-Targeting drug help friedreich ataxia patients see better?

NCT ID NCT05168774

First seen Jan 05, 2026 · Last updated May 15, 2026 · Updated 24 times

Summary

This early-phase study tested the safety and potential benefits of elamipretide for treating vision loss in people with Friedreich ataxia (FRDA). Twenty participants with advanced symptoms received either a low or high dose of the drug. The main goal was to see if it improved high-contrast visual acuity, with secondary measures including low-contrast vision and other visual functions.

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Contacts and locations

Locations

  • Children's Hospital of Philadelphia - Neurology

    Philadelphia, Pennsylvania, 19104, United States

Conditions

Explore the condition pages connected to this study.