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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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Children's Hospital of Philadelphia - Neurology
Philadelphia, Pennsylvania, 19104, United States
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