Gene therapy targets heart failure in rare genetic disease
NCT ID NCT05445323
First seen Jan 04, 2026 · Last updated May 23, 2026 · Updated 16 times
Summary
This early-stage study tests a one-time gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The therapy delivers a working copy of the frataxin gene directly to heart cells to improve heart function. Researchers will monitor safety and heart changes over 5 years.
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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.
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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Contacts and locations
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Locations
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Ataxia Center and HD Center of Excellence, University of California
Los Angeles, California, 90095, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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University of South Florida
Tampa, Florida, 33612, United States
Conditions
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